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Volumn 56, Issue 3, 2008, Pages 236-247

Duchenne muscular dystrophy

Author keywords

Continuous positive airway pressure; Corticosteroids; Creatine kinase; Duchenne muscular dystrophy; Gene therapy; Muscle disease; Pediatric

Indexed keywords

ADENOVIRUS VECTOR; ANTISENSE OLIGONUCLEOTIDE; AZATHIOPRINE; BETA ADRENERGIC RECEPTOR BLOCKING AGENT; CREATINE KINASE; CYCLOSPORIN; DEFLAZACORT; DIPEPTIDYL CARBOXYPEPTIDASE INHIBITOR; GENTAMICIN; LAXATIVE; OXANDROLONE; PREDNISOLONE; PREDNISONE; PTC 124; UTROPHIN;

EID: 56049106835     PISSN: 00283886     EISSN: 19984022     Source Type: Journal    
DOI: 10.4103/0028-3886.43441     Document Type: Review
Times cited : (63)

References (190)
  • 1
    • 0020082152 scopus 로고
    • Prevalence and incidence of muscular dystrophy in Alberta, Canada
    • Monckton G, Hoskin V, Warren S. Prevalence and incidence of muscular dystrophy in Alberta, Canada. Clin Genet 1982;21:19-24.
    • (1982) Clin Genet , vol.21 , pp. 19-24
    • Monckton, G.1    Hoskin, V.2    Warren, S.3
  • 2
    • 0038305426 scopus 로고    scopus 로고
    • Neuromuscular disorders of infancy, childhood and adolescence
    • Oxford: Butterworth- Heinemann;
    • Jones H, De Vivo DC, Darras BT. Neuromuscular disorders of infancy, childhood and adolescence. A clinician's approach. Oxford: Butterworth- Heinemann; 2003.
    • (2003) A clinician's approach
    • Jones, H.1    De Vivo, D.C.2    Darras, B.T.3
  • 3
    • 0021361231 scopus 로고
    • Cardiac rhythm and conduction in Duchenne's muscular dystrophy: A prospective study of 20 patients
    • Perloff JK. Cardiac rhythm and conduction in Duchenne's muscular dystrophy: A prospective study of 20 patients. J Am Coll Cardiol 1984;3:1263-8.
    • (1984) J Am Coll Cardiol , vol.3 , pp. 1263-1268
    • Perloff, J.K.1
  • 5
    • 0025017751 scopus 로고
    • The incidence and evolution of cardiomyopathy in Duchenne muscular dystrophy
    • Nigro G, Comi LI, Politano L, Bain RJ. The incidence and evolution of cardiomyopathy in Duchenne muscular dystrophy. Int J Cardiol 1990;26:271-7.
    • (1990) Int J Cardiol , vol.26 , pp. 271-277
    • Nigro, G.1    Comi, L.I.2    Politano, L.3    Bain, R.J.4
  • 6
    • 0037114204 scopus 로고    scopus 로고
    • Changes in spirometry over time as a prognostic marker in patients with Duchenne muscular dystrophy
    • Phillips MF, Quinlivan RC, Edwards RH, Calverley PM. Changes in spirometry over time as a prognostic marker in patients with Duchenne muscular dystrophy. Am J Respir Crit Care Med 2001;164:2191-4.
    • (2001) Am J Respir Crit Care Med , vol.164 , pp. 2191-2194
    • Phillips, M.F.1    Quinlivan, R.C.2    Edwards, R.H.3    Calverley, P.M.4
  • 7
    • 0019432012 scopus 로고
    • Respiratory function in the muscular dystrophies
    • Rideau Y, Jankowski LW, Grellet J. Respiratory function in the muscular dystrophies. Muscle Nerve 1981;4:155-64.
    • (1981) Muscle Nerve , vol.4 , pp. 155-164
    • Rideau, Y.1    Jankowski, L.W.2    Grellet, J.3
  • 8
    • 0025317772 scopus 로고    scopus 로고
    • Baydur A, Gilgoff I, Prentice W, Carlson M, Fischer DA. Decline in respiratory function and experience with long-term assisted ventilation in advanced Duchenne's muscular dystrophy. Chest 1990;97:884-9.
    • Baydur A, Gilgoff I, Prentice W, Carlson M, Fischer DA. Decline in respiratory function and experience with long-term assisted ventilation in advanced Duchenne's muscular dystrophy. Chest 1990;97:884-9.
  • 9
    • 0015988206 scopus 로고
    • Pulmonary function in Duchenne muscular dystrophy related to stage of disease
    • Inkley SR, Oldenburg FC, Vignos PJ Jr. Pulmonary function in Duchenne muscular dystrophy related to stage of disease. Am J Med 1974;56:297-306.
    • (1974) Am J Med , vol.56 , pp. 297-306
    • Inkley, S.R.1    Oldenburg, F.C.2    Vignos Jr., P.J.3
  • 10
    • 33644816152 scopus 로고    scopus 로고
    • Sleep-related breathing disorder in Duchenne muscular dystrophy: Disease spectrum in the paediatric population
    • Suresh S, Wales P, Dakin C, Harris MA, Cooper DG. Sleep-related breathing disorder in Duchenne muscular dystrophy: Disease spectrum in the paediatric population. J Paediatr Child Health 2005;41:500-3.
    • (2005) J Paediatr Child Health , vol.41 , pp. 500-503
    • Suresh, S.1    Wales, P.2    Dakin, C.3    Harris, M.A.4    Cooper, D.G.5
  • 11
    • 0036344202 scopus 로고    scopus 로고
    • Patterns and predictors of sleep disordered breathing in primary myopathies
    • Ragette R, Mellies U, Schwake C, Voit T, Teschler H. Patterns and predictors of sleep disordered breathing in primary myopathies. Thorax 2002;57:724-8.
    • (2002) Thorax , vol.57 , pp. 724-728
    • Ragette, R.1    Mellies, U.2    Schwake, C.3    Voit, T.4    Teschler, H.5
  • 12
    • 0033954585 scopus 로고    scopus 로고
    • Daytime predictors of sleep hypoventilation in Duchenne muscular dystrophy
    • Hukins CA, Hillman DR. Daytime predictors of sleep hypoventilation in Duchenne muscular dystrophy. Am J Respir Crit Care Med 2000;161:166-70.
    • (2000) Am J Respir Crit Care Med , vol.161 , pp. 166-170
    • Hukins, C.A.1    Hillman, D.R.2
  • 13
    • 0031759227 scopus 로고    scopus 로고
    • Impact of nasal ventilation on survival in hypercapnic Duchenne muscular dystrophy
    • Simonds AK, Muntoni F, Heather S, Fielding S. Impact of nasal ventilation on survival in hypercapnic Duchenne muscular dystrophy. Thorax 1998;53:949-52.
    • (1998) Thorax , vol.53 , pp. 949-952
    • Simonds, A.K.1    Muntoni, F.2    Heather, S.3    Fielding, S.4
  • 14
    • 35348967896 scopus 로고    scopus 로고
    • Mechanical ventilation in Duchenne patients with chronic respiratory insufficiency: Clinical implications of 20 years published experience
    • Toussaint M, Chatwin M, Soudon P. Mechanical ventilation in Duchenne patients with chronic respiratory insufficiency: Clinical implications of 20 years published experience. Chron 2007;4:167-77.
    • (2007) Chron , vol.4 , pp. 167-177
    • Toussaint, M.1    Chatwin, M.2    Soudon, P.3
  • 15
    • 0028127753 scopus 로고
    • Long-term nasal intermittent positive pressure ventilation in advanced Duchenne's muscular dystrophy
    • Vianello A, Bevilacqua M, Salvador V, Cardaioli C, Vincenti E. Long-term nasal intermittent positive pressure ventilation in advanced Duchenne's muscular dystrophy. Chest 1994;105:445-8.
    • (1994) Chest , vol.105 , pp. 445-448
    • Vianello, A.1    Bevilacqua, M.2    Salvador, V.3    Cardaioli, C.4    Vincenti, E.5
  • 16
    • 0019390636 scopus 로고
    • Intellect and behaviour in Duchenne muscular dystrophy
    • Leibowitz D, Dubowitz V. Intellect and behaviour in Duchenne muscular dystrophy. Dev Med Child Neurol 1981;23:577-90.
    • (1981) Dev Med Child Neurol , vol.23 , pp. 577-590
    • Leibowitz, D.1    Dubowitz, V.2
  • 17
    • 0034933730 scopus 로고    scopus 로고
    • Intelligence and Duchenne muscular dystrophy: Full-scale, verbal and performance intelligence quotients
    • Cotton S, Voudouris NJ, Greenwood KM. Intelligence and Duchenne muscular dystrophy: Full-scale, verbal and performance intelligence quotients. Dev Med Child Neurol 2001;43:497-501.
    • (2001) Dev Med Child Neurol , vol.43 , pp. 497-501
    • Cotton, S.1    Voudouris, N.J.2    Greenwood, K.M.3
  • 18
    • 0036156243 scopus 로고    scopus 로고
    • Brain function in Duchenne muscular dystrophy
    • Anderson JL, Head SI, Rae C, Morley JW. Brain function in Duchenne muscular dystrophy. Brain 2002;125:4-13.
    • (2002) Brain , vol.125 , pp. 4-13
    • Anderson, J.L.1    Head, S.I.2    Rae, C.3    Morley, J.W.4
  • 19
    • 0018831758 scopus 로고
    • Analysis of verbal disability in Duchenne muscular dystrophy
    • Karagan NJ, Richman LC, Sorensen JP. Analysis of verbal disability in Duchenne muscular dystrophy. J Nerv Ment Dis 1980;168:419-23.
    • (1980) J Nerv Ment Dis , vol.168 , pp. 419-423
    • Karagan, N.J.1    Richman, L.C.2    Sorensen, J.P.3
  • 20
    • 0018448516 scopus 로고
    • Intellectual functioning in Duchenne muscular dystrophy: A review
    • Karagan NJ. Intellectual functioning in Duchenne muscular dystrophy: A review. Psychol Bull 1979;86:250-9.
    • (1979) Psychol Bull , vol.86 , pp. 250-259
    • Karagan, N.J.1
  • 21
    • 0008023765 scopus 로고
    • Mental retardation in association with progressive dystrophy
    • Allen JE, Rodgin DW. Mental retardation in association with progressive dystrophy. Am J Dis Child 1960;100:208-11.
    • (1960) Am J Dis Child , vol.100 , pp. 208-211
    • Allen, J.E.1    Rodgin, D.W.2
  • 22
    • 31144459504 scopus 로고    scopus 로고
    • Association of Duchenne muscular dystrophy with autism spectrum disorder
    • Wu JY, Kuban KC, Allred E, Shapiro F, Darras BT. Association of Duchenne muscular dystrophy with autism spectrum disorder. J Child Neurol 2005;20:790-5.
    • (2005) J Child Neurol , vol.20 , pp. 790-795
    • Wu, J.Y.1    Kuban, K.C.2    Allred, E.3    Shapiro, F.4    Darras, B.T.5
  • 23
    • 42449160246 scopus 로고    scopus 로고
    • Neuropsychiatric disorders in males with duchenne muscular dystrophy: Frequency rate of attention-deficit hyperactivity disorder (ADHD), autism spectrum disorder, and obsessive - compulsive disorder
    • Hendriksen JG, Vles JS. Neuropsychiatric disorders in males with duchenne muscular dystrophy: Frequency rate of attention-deficit hyperactivity disorder (ADHD), autism spectrum disorder, and obsessive - compulsive disorder. J Child Neurol 2008;23:477-81.
    • (2008) J Child Neurol , vol.23 , pp. 477-481
    • Hendriksen, J.G.1    Vles, J.S.2
  • 24
    • 0024708810 scopus 로고
    • Progression of scoliosis in Duchenne muscular dystrophy
    • Smith AD, Koreska J, Moseley CF. Progression of scoliosis in Duchenne muscular dystrophy. J Bone Joint Surg Am 1989;71:1066-74.
    • (1989) J Bone Joint Surg Am , vol.71 , pp. 1066-1074
    • Smith, A.D.1    Koreska, J.2    Moseley, C.F.3
  • 25
    • 0023768755 scopus 로고
    • Prevention of rapidly progressive scoliosis in Duchenne muscular dystrophy by prolongation of walking with orthoses
    • Rodillo EB, Fernandez-Bermejo E, Heckmatt JZ, Dubowitz V. Prevention of rapidly progressive scoliosis in Duchenne muscular dystrophy by prolongation of walking with orthoses. J Child Neurol 1988;3:269-74.
    • (1988) J Child Neurol , vol.3 , pp. 269-274
    • Rodillo, E.B.1    Fernandez-Bermejo, E.2    Heckmatt, J.Z.3    Dubowitz, V.4
  • 27
    • 0033962313 scopus 로고    scopus 로고
    • Bone mineral density and fractures in boys with Duchenne muscular dystrophy
    • Larson CM, Henderson RC. Bone mineral density and fractures in boys with Duchenne muscular dystrophy. J Pediatr Orthop 2000;20:71-4.
    • (2000) J Pediatr Orthop , vol.20 , pp. 71-74
    • Larson, C.M.1    Henderson, R.C.2
  • 30
    • 0036215815 scopus 로고    scopus 로고
    • Decreased bone density in ambulatory patients with duchenne muscular dystrophy
    • Aparicio LF, Jurkovic M, DeLullo J. Decreased bone density in ambulatory patients with duchenne muscular dystrophy. J Pediatr Orthop 2002;22:179-81.
    • (2002) J Pediatr Orthop , vol.22 , pp. 179-181
    • Aparicio, L.F.1    Jurkovic, M.2    DeLullo, J.3
  • 31
    • 0020663030 scopus 로고
    • Malignant hyperthermia in a child with Duchenne muscular dystrophy
    • Kelfer HM, Singer WD, Reynolds RN. Malignant hyperthermia in a child with Duchenne muscular dystrophy. Pediatrics 1983;71:118-9.
    • (1983) Pediatrics , vol.71 , pp. 118-119
    • Kelfer, H.M.1    Singer, W.D.2    Reynolds, R.N.3
  • 32
    • 0026458101 scopus 로고
    • Malignant hyperthermia and neuromuscular disease
    • Wedel DJ. Malignant hyperthermia and neuromuscular disease. Neuromuscul Disord 1992;2:157-64.
    • (1992) Neuromuscul Disord , vol.2 , pp. 157-164
    • Wedel, D.J.1
  • 33
    • 0022536395 scopus 로고
    • Duchenne muscular dystrophy and malignant hyperthermia-two case reports
    • Wang JM, Stanley TH. Duchenne muscular dystrophy and malignant hyperthermia-two case reports. Can Anaesth Soc J 1986;33:492-7.
    • (1986) Can Anaesth Soc J , vol.33 , pp. 492-497
    • Wang, J.M.1    Stanley, T.H.2
  • 34
    • 37749041384 scopus 로고    scopus 로고
    • Duchenne muscular dystrophy: An old anesthesia problem revisited
    • Hayes J, Veyckemans F, Bissonnette B. Duchenne muscular dystrophy: An old anesthesia problem revisited. Paediatr Anaesth 2008;18:100-6.
    • (2008) Paediatr Anaesth , vol.18 , pp. 100-106
    • Hayes, J.1    Veyckemans, F.2    Bissonnette, B.3
  • 35
    • 0023614271 scopus 로고
    • Complete cloning of the Duchenne muscular dystrophy (DMD) cDNA and preliminary genomic organization of the DMD gene in normal and affected individuals
    • Koenig M, Hoffman EP, Bertelson CJ, Monaco AP, Feener C, Kunkel LM. Complete cloning of the Duchenne muscular dystrophy (DMD) cDNA and preliminary genomic organization of the DMD gene in normal and affected individuals. Cell 1987;50:509-17.
    • (1987) Cell , vol.50 , pp. 509-517
    • Koenig, M.1    Hoffman, E.P.2    Bertelson, C.J.3    Monaco, A.P.4    Feener, C.5    Kunkel, L.M.6
  • 37
    • 0024314433 scopus 로고
    • Molecular genetics of Duchenne and Becker muscular dystrophy: Emphasis on improved diagnosis
    • Kunkel LM, Beggs AH, Hoffman EP. Molecular genetics of Duchenne and Becker muscular dystrophy: Emphasis on improved diagnosis. Clin Chem 1989;35:B21-4.
    • (1989) Clin Chem , vol.35
    • Kunkel, L.M.1    Beggs, A.H.2    Hoffman, E.P.3
  • 38
    • 0024370782 scopus 로고
    • Dystrophin. The gene and its product
    • Mandel JL. Dystrophin. The gene and its product. Nature 1989;339:584-6.
    • (1989) Nature , vol.339 , pp. 584-586
    • Mandel, J.L.1
  • 39
    • 0023614188 scopus 로고
    • Dystrophin: The protein product of the Duchenne muscular dystrophy locus
    • Hoffman EP, Brown RH Jr, Kunkel LM. Dystrophin: The protein product of the Duchenne muscular dystrophy locus. Cell 1987;51:919-28.
    • (1987) Cell , vol.51 , pp. 919-928
    • Hoffman, E.P.1    Brown Jr, R.H.2    Kunkel, L.M.3
  • 40
    • 0023925292 scopus 로고
    • The Duchenne muscular dystrophy gene product is localized in sarcolemma of human skeletal muscle
    • Zubrzycka-Gaarn EE, Bulman DE, Karpati G, Burghes AH, Belfall B, Klamut HJ, et al. The Duchenne muscular dystrophy gene product is localized in sarcolemma of human skeletal muscle. Nature 1988;333:466-9.
    • (1988) Nature , vol.333 , pp. 466-469
    • Zubrzycka-Gaarn, E.E.1    Bulman, D.E.2    Karpati, G.3    Burghes, A.H.4    Belfall, B.5    Klamut, H.J.6
  • 41
    • 0025272250 scopus 로고
    • Deficiency of a glycoprotein component of the dystrophin complex in dystrophic muscle
    • Ervasti JM, Ohlendieck K, Kahl SD, Gaver MG, Campbell KP. Deficiency of a glycoprotein component of the dystrophin complex in dystrophic muscle. Nature 1990;345:315-9.
    • (1990) Nature , vol.345 , pp. 315-319
    • Ervasti, J.M.1    Ohlendieck, K.2    Kahl, S.D.3    Gaver, M.G.4    Campbell, K.P.5
  • 42
    • 0035190381 scopus 로고    scopus 로고
    • The dystrophin-glycoprotein complex, cellular signaling, and the regulation of cell survival in the muscular dystrophies
    • Rando TA. The dystrophin-glycoprotein complex, cellular signaling, and the regulation of cell survival in the muscular dystrophies. Muscle Nerve 2001;24:1575-94.
    • (2001) Muscle Nerve , vol.24 , pp. 1575-1594
    • Rando, T.A.1
  • 43
    • 0023904860 scopus 로고
    • The complete sequence of dystrophin predicts a rod-shaped cytoskeletal protein
    • Koenig M, Monaco AP, Kunkel LM. The complete sequence of dystrophin predicts a rod-shaped cytoskeletal protein. Cell 1988;53:219-28.
    • (1988) Cell , vol.53 , pp. 219-228
    • Koenig, M.1    Monaco, A.P.2    Kunkel, L.M.3
  • 44
    • 0032915455 scopus 로고    scopus 로고
    • The WW domain of dystrophin requires EF-hands region to interact with beta-dystroglycan
    • Rentschler S, Linn H, Deininger K, Bedford MT, Espanel X, Sudol M. The WW domain of dystrophin requires EF-hands region to interact with beta-dystroglycan. Biol Chem 1999;380:431-42.
    • (1999) Biol Chem , vol.380 , pp. 431-442
    • Rentschler, S.1    Linn, H.2    Deininger, K.3    Bedford, M.T.4    Espanel, X.5    Sudol, M.6
  • 45
    • 0024600620 scopus 로고
    • Association of dystrophin and an integral membrane glycoprotein
    • Campbell KP, Kahl SD. Association of dystrophin and an integral membrane glycoprotein. Nature 1989;338:259-62.
    • (1989) Nature , vol.338 , pp. 259-262
    • Campbell, K.P.1    Kahl, S.D.2
  • 46
    • 0027470203 scopus 로고
    • The structural and functional diversity of dystrophin
    • Ahn AH, Kunkel LM. The structural and functional diversity of dystrophin. Nat Genet 1993;3:283-91.
    • (1993) Nat Genet , vol.3 , pp. 283-291
    • Ahn, A.H.1    Kunkel, L.M.2
  • 47
    • 0023718118 scopus 로고
    • An explanation for the phenotypic differences between patients bearing partial deletions of the DMD locus
    • Monaco AP, Bertelson CJ, Liechti-Gallati S, Moser H, Kunkel LM. An explanation for the phenotypic differences between patients bearing partial deletions of the DMD locus. Genomics 1988;2:90-5.
    • (1988) Genomics , vol.2 , pp. 90-95
    • Monaco, A.P.1    Bertelson, C.J.2    Liechti-Gallati, S.3    Moser, H.4    Kunkel, L.M.5
  • 48
    • 33746766278 scopus 로고    scopus 로고
    • Entries in the Leiden Duchenne muscular dystrophy mutation database: An overview of mutation types and paradoxical cases that confirm the reading-frame rule
    • Aartsma-Rus A, Van Deutekom JC, Fokkema IF, Van Ommen GJ, Den Dunnen JT. Entries in the Leiden Duchenne muscular dystrophy mutation database: An overview of mutation types and paradoxical cases that confirm the reading-frame rule. Muscle Nerve 2006;34:135-44.
    • (2006) Muscle Nerve , vol.34 , pp. 135-144
    • Aartsma-Rus, A.1    Van Deutekom, J.C.2    Fokkema, I.F.3    Van Ommen, G.J.4    Den Dunnen, J.T.5
  • 49
    • 0344420060 scopus 로고    scopus 로고
    • Dystrophin and mutations: One gene, several proteins, multiple phenotypes
    • Muntoni F, Torelli S, Ferlini A. Dystrophin and mutations: One gene, several proteins, multiple phenotypes. Lancet neurol 2003;2:731-40.
    • (2003) Lancet neurol , vol.2 , pp. 731-740
    • Muntoni, F.1    Torelli, S.2    Ferlini, A.3
  • 50
    • 0025244924 scopus 로고
    • Detection of 98% of DMD/BMD gene deletions by polymerase chain reaction
    • Beggs AH, Koenig M, Boyce FM, Kunkel LM. Detection of 98% of DMD/BMD gene deletions by polymerase chain reaction. Hum Genet 1990;86:45-8.
    • (1990) Hum Genet , vol.86 , pp. 45-48
    • Beggs, A.H.1    Koenig, M.2    Boyce, F.M.3    Kunkel, L.M.4
  • 53
    • 0028303798 scopus 로고    scopus 로고
    • Roberts RG, Gardner RJ, Bobrow M. Searching for the 1 in 2,400,000: A review of dystrophin gene point mutations. Hum Mutat 1994;4:1-11.
    • Roberts RG, Gardner RJ, Bobrow M. Searching for the 1 in 2,400,000: A review of dystrophin gene point mutations. Hum Mutat 1994;4:1-11.
  • 54
    • 0031908112 scopus 로고    scopus 로고
    • The molecular basis of activity-induced muscle injury in Duchenne muscular dystrophy
    • Petrof BJ. The molecular basis of activity-induced muscle injury in Duchenne muscular dystrophy. Mol Cell Biochem 1998;179:111-23.
    • (1998) Mol Cell Biochem , vol.179 , pp. 111-123
    • Petrof, B.J.1
  • 55
    • 33947258069 scopus 로고    scopus 로고
    • Pathophysiology of duchenne muscular dystrophy: Current hypotheses
    • Deconinck N, Dan B. Pathophysiology of duchenne muscular dystrophy: Current hypotheses. Pediatr Neurol 2007;36:1-7.
    • (2007) Pediatr Neurol , vol.36 , pp. 1-7
    • Deconinck, N.1    Dan, B.2
  • 56
    • 0025018325 scopus 로고
    • Dystrophin analysis in Duchenne and Becker muscular dystrophy carriers: Correlation with intracellular calcium and albumin
    • Morandi L, Mora M, Gussoni E, Tedeschi S, Cornelio F. Dystrophin analysis in Duchenne and Becker muscular dystrophy carriers: Correlation with intracellular calcium and albumin. Ann Neurol 1990;28:674-9.
    • (1990) Ann Neurol , vol.28 , pp. 674-679
    • Morandi, L.1    Mora, M.2    Gussoni, E.3    Tedeschi, S.4    Cornelio, F.5
  • 57
    • 0017797971 scopus 로고
    • Intracellular calcium accumulation in Duchenne dystrophy and other myopathies: A study of 567,000 muscle fibers in 114 biopsies
    • Bodensteiner JB, Engel AG. Intracellular calcium accumulation in Duchenne dystrophy and other myopathies: A study of 567,000 muscle fibers in 114 biopsies. Neurology 1978;28:439-46.
    • (1978) Neurology , vol.28 , pp. 439-446
    • Bodensteiner, J.B.1    Engel, A.G.2
  • 58
    • 0035878751 scopus 로고    scopus 로고
    • Calcium currents and transients in co-cultured contracting normal and Duchenne muscular dystrophy human myotubes
    • Imbert N, Vandebrouck C, Duport G, Raymond G, Hassoni AA, Constantin B, et al. Calcium currents and transients in co-cultured contracting normal and Duchenne muscular dystrophy human myotubes. J Physiol 2001;534:343-55.
    • (2001) J Physiol , vol.534 , pp. 343-355
    • Imbert, N.1    Vandebrouck, C.2    Duport, G.3    Raymond, G.4    Hassoni, A.A.5    Constantin, B.6
  • 59
    • 0029149471 scopus 로고
    • Nitric oxide synthase complexed with dystrophin and absent from skeletal muscle sarcolemma in Duchenne muscular dystrophy
    • Brenman JE, Chao DS, Xia H, Aldape K, Bredt DS. Nitric oxide synthase complexed with dystrophin and absent from skeletal muscle sarcolemma in Duchenne muscular dystrophy. Cell 1995;82:743-52.
    • (1995) Cell , vol.82 , pp. 743-752
    • Brenman, J.E.1    Chao, D.S.2    Xia, H.3    Aldape, K.4    Bredt, D.S.5
  • 60
    • 0034610326 scopus 로고    scopus 로고
    • Functional muscle ischemia in neuronal nitric oxide synthase-deficient skeletal muscle of children with Duchenne muscular dystrophy
    • Sander M, Chavoshan B, Harris SA, Iannaccone ST, Stull JT, Thomas GD, et al. Functional muscle ischemia in neuronal nitric oxide synthase-deficient skeletal muscle of children with Duchenne muscular dystrophy. Proc Natl Acad Sci USA 2000;97:13818-23.
    • (2000) Proc Natl Acad Sci USA , vol.97 , pp. 13818-13823
    • Sander, M.1    Chavoshan, B.2    Harris, S.A.3    Iannaccone, S.T.4    Stull, J.T.5    Thomas, G.D.6
  • 62
    • 0042011472 scopus 로고    scopus 로고
    • Novel therapies for Duchenne muscular dystrophy
    • Kapsa R, Kornberg AJ, Byrne E. Novel therapies for Duchenne muscular dystrophy. Lancet Neurol 2003;2:299-310.
    • (2003) Lancet Neurol , vol.2 , pp. 299-310
    • Kapsa, R.1    Kornberg, A.J.2    Byrne, E.3
  • 64
    • 0024850505 scopus 로고
    • Serum enzymes in disease of skeletal muscle
    • Rosalki SB. Serum enzymes in disease of skeletal muscle. Clin Lab Med 1989;9:767-81.
    • (1989) Clin Lab Med , vol.9 , pp. 767-781
    • Rosalki, S.B.1
  • 65
    • 0020522923 scopus 로고
    • Clinical investigation in Duchenne dystrophy: 2, Determination of the "power" of therapeutic trials based on the natural history
    • Brooke MH, Fenichel GM, Griggs RC, Mendell JR, Moxley R, Miller JP, et al. Clinical investigation in Duchenne dystrophy: 2, Determination of the "power" of therapeutic trials based on the natural history. Muscle Nerve 1983;6:91-103.
    • (1983) Muscle Nerve , vol.6 , pp. 91-103
    • Brooke, M.H.1    Fenichel, G.M.2    Griggs, R.C.3    Mendell, J.R.4    Moxley, R.5    Miller, J.P.6
  • 66
    • 0025801496 scopus 로고
    • Serum creatine-kinase (CK) and pyruvate-kinase (PK) activities in Duchenne (DMD) as compared with Becker (BMD) muscular dystrophy
    • Zatz M, Rapaport D, Vainzof M, Passos-Bueno MR, Bortolini ER, Pavanello Rde C, et al. Serum creatine-kinase (CK) and pyruvate-kinase (PK) activities in Duchenne (DMD) as compared with Becker (BMD) muscular dystrophy. J Neurol Sci 1991;102:190-6.
    • (1991) J Neurol Sci , vol.102 , pp. 190-196
    • Zatz, M.1    Rapaport, D.2    Vainzof, M.3    Passos-Bueno, M.R.4    Bortolini, E.R.5    Pavanello Rde, C.6
  • 67
    • 0034332433 scopus 로고    scopus 로고
    • Tay SK, Ong HT, Low PS. Transaminitis in Duchenne's muscular dystrophy. Ann Acad Med Singapore 2000;29:719-22.
    • Tay SK, Ong HT, Low PS. Transaminitis in Duchenne's muscular dystrophy. Ann Acad Med Singapore 2000;29:719-22.
  • 68
    • 0027458614 scopus 로고
    • Diagnosis of occult muscular dystrophy: Importance of the "chance" finding of elevated serum aminotransferase activities
    • Morse RP, Rosman NP. Diagnosis of occult muscular dystrophy: Importance of the "chance" finding of elevated serum aminotransferase activities. J Pediatr 1993;122:254-6.
    • (1993) J Pediatr , vol.122 , pp. 254-256
    • Morse, R.P.1    Rosman, N.P.2
  • 69
    • 0015935012 scopus 로고
    • Serum enzyme alterations in neuromuscular disorders
    • Munsat TL, Baloh R, Pearson CM, Fowler W Jr. Serum enzyme alterations in neuromuscular disorders. JAMA 1973;226:1536-43.
    • (1973) JAMA , vol.226 , pp. 1536-1543
    • Munsat, T.L.1    Baloh, R.2    Pearson, C.M.3    Fowler Jr., W.4
  • 70
    • 0018920710 scopus 로고
    • Biochemistry of muscle membranes in Duchenne muscular dystrophy
    • Rowland LP. Biochemistry of muscle membranes in Duchenne muscular dystrophy. Muscle Nerve 1980;3:3-20.
    • (1980) Muscle Nerve , vol.3 , pp. 3-20
    • Rowland, L.P.1
  • 71
    • 0035188991 scopus 로고    scopus 로고
    • Liver function tests are not always tests of liver function
    • Korones DN, Brown MR, Palis J. "Liver function tests" are not always tests of liver function. Am J Hematol 2001;66:46-8.
    • (2001) Am J Hematol , vol.66 , pp. 46-48
    • Korones, D.N.1    Brown, M.R.2    Palis, J.3
  • 72
    • 0021318746 scopus 로고
    • The enzymology of skeletal muscle disorders
    • Lott JA, Landesman PW. The enzymology of skeletal muscle disorders. Crit Rev Clin Lab Sci 1984;20:153-90.
    • (1984) Crit Rev Clin Lab Sci , vol.20 , pp. 153-190
    • Lott, J.A.1    Landesman, P.W.2
  • 73
    • 0016819081 scopus 로고
    • Relationship of spontaneous fibrillation potentials to muscle fibre segmentation in human muscular dystrophy
    • Desmedt JE, Borenstein S. Relationship of spontaneous fibrillation potentials to muscle fibre segmentation in human muscular dystrophy. Nature 1975;258:531-4.
    • (1975) Nature , vol.258 , pp. 531-534
    • Desmedt, J.E.1    Borenstein, S.2
  • 74
    • 0017141250 scopus 로고
    • Regeneration in Duchenne muscular dystrophy: Electromyographic evidence
    • Desmedt JE, Borenstein S. Regeneration in Duchenne muscular dystrophy: Electromyographic evidence. Arch Neurol 1976;33:642-50.
    • (1976) Arch Neurol , vol.33 , pp. 642-650
    • Desmedt, J.E.1    Borenstein, S.2
  • 75
    • 0035964237 scopus 로고    scopus 로고
    • a muscle biopsy in Duchenne dystrophy really necessary?
    • Muntoni F. Is a muscle biopsy in Duchenne dystrophy really necessary? Neurology 2001;57:574-5.
    • (2001) Neurology , vol.57 , pp. 574-575
    • Is, M.F.1
  • 76
    • 0014127574 scopus 로고    scopus 로고
    • Bell CD, Conen PE. Change in fiber size in Duchenne muscular dystrophy. Neurology 1967;17:902-13.
    • Bell CD, Conen PE. Change in fiber size in Duchenne muscular dystrophy. Neurology 1967;17:902-13.
  • 77
    • 0026063851 scopus 로고
    • Differentiation of Duchenne and Becker muscular dystrophy phenotypes with amino and carboxy-terminal antisera specific for dystrophin
    • Bulman DE, Murphy EG, Zubrzycka-Gaarn EE, Worton RG, Ray PN. Differentiation of Duchenne and Becker muscular dystrophy phenotypes with amino and carboxy-terminal antisera specific for dystrophin. Am J Hum Genet 1991;48:295-304.
    • (1991) Am J Hum Genet , vol.48 , pp. 295-304
    • Bulman, D.E.1    Murphy, E.G.2    Zubrzycka-Gaarn, E.E.3    Worton, R.G.4    Ray, P.N.5
  • 78
    • 0025822392 scopus 로고
    • Dystrophin as a diagnostic marker in Duchenne and Becker muscular dystrophy: Correlation of immunofluorescence and western blot
    • Voit T, Stuettgen P, Cremer M, Goebel HH. Dystrophin as a diagnostic marker in Duchenne and Becker muscular dystrophy: Correlation of immunofluorescence and western blot. Neuropediatrics 1991;22:152-62.
    • (1991) Neuropediatrics , vol.22 , pp. 152-162
    • Voit, T.1    Stuettgen, P.2    Cremer, M.3    Goebel, H.H.4
  • 80
    • 0027203989 scopus 로고
    • Integrated study of 100 patients with Xp21 linked muscular dystrophy using clinical, genetic, immunochemical, and histopathological data: Part 1, Trends across the clinical groups
    • Nicholson LV, Johnson MA, Bushby KM, Gardner-Medwin D, Curtis A, Ginjaar IB, et al. Integrated study of 100 patients with Xp21 linked muscular dystrophy using clinical, genetic, immunochemical, and histopathological data: Part 1, Trends across the clinical groups. J Med Genet 1993;30:728-36.
    • (1993) J Med Genet , vol.30 , pp. 728-736
    • Nicholson, L.V.1    Johnson, M.A.2    Bushby, K.M.3    Gardner-Medwin, D.4    Curtis, A.5    Ginjaar, I.B.6
  • 81
    • 0023906647 scopus 로고
    • Characterization of dystrophin in muscle-biopsy specimens from patients with Duchenne's or Becker's muscular dystrophy
    • Hoffman EP, Fischbeck KH, Brown RH, Johnson M, Medori R, Loike JD, et al. Characterization of dystrophin in muscle-biopsy specimens from patients with Duchenne's or Becker's muscular dystrophy. N Engl J Med 1988;318:1363-8.
    • (1988) N Engl J Med , vol.318 , pp. 1363-1368
    • Hoffman, E.P.1    Fischbeck, K.H.2    Brown, R.H.3    Johnson, M.4    Medori, R.5    Loike, J.D.6
  • 82
    • 0024245082 scopus 로고
    • Deletion screening of the Duchenne muscular dystrophy locus via multiplex DNA amplification
    • Chamberlain JS, Gibbs RA, Ranier JE, Nguyen PN, Caskey CT. Deletion screening of the Duchenne muscular dystrophy locus via multiplex DNA amplification. Nucleic Acids Res 1988;16:11141-56.
    • (1988) Nucleic Acids Res , vol.16 , pp. 11141-11156
    • Chamberlain, J.S.1    Gibbs, R.A.2    Ranier, J.E.3    Nguyen, P.N.4    Caskey, C.T.5
  • 83
    • 11444268506 scopus 로고    scopus 로고
    • Improved molecular diagnosis of dystrophin gene mutations using the multiplex ligation-dependent probe amplification method
    • Schwartz M, Duno M. Improved molecular diagnosis of dystrophin gene mutations using the multiplex ligation-dependent probe amplification method. Genet Test 2004;8:361-7.
    • (2004) Genet Test , vol.8 , pp. 361-367
    • Schwartz, M.1    Duno, M.2
  • 85
    • 15444370412 scopus 로고    scopus 로고
    • MLPA analysis for the detection of deletions, duplications and complex rearrangements in the dystrophin gene: Potential and pitfalls
    • Janssen B, Hartmann C, Scholz V, Jauch A, Zschocke J. MLPA analysis for the detection of deletions, duplications and complex rearrangements in the dystrophin gene: Potential and pitfalls. Neurogenetics 2005;6:29-35.
    • (2005) Neurogenetics , vol.6 , pp. 29-35
    • Janssen, B.1    Hartmann, C.2    Scholz, V.3    Jauch, A.4    Zschocke, J.5
  • 86
    • 20344366588 scopus 로고    scopus 로고
    • Identification of deletions and duplications of the DMD gene in affected males and carrier females by multiple ligation probe amplification (MLPA)
    • Gatta V, Scarciolla O, Gaspari AR, Palka C, De Angelis MV, Di Muzio A, et al. Identification of deletions and duplications of the DMD gene in affected males and carrier females by multiple ligation probe amplification (MLPA). Hum Genet 2005;117:92-8.
    • (2005) Hum Genet , vol.117 , pp. 92-98
    • Gatta, V.1    Scarciolla, O.2    Gaspari, A.R.3    Palka, C.4    De Angelis, M.V.5    Di Muzio, A.6
  • 88
    • 2942523954 scopus 로고    scopus 로고
    • Detection of mutations in the dystrophin gene via automated DHPLC screening and direct sequencing
    • Bennett RR, den Dunnen J, O'Brien KF, Darras BT, Kunkel LM. Detection of mutations in the dystrophin gene via automated DHPLC screening and direct sequencing. BMC Genet 2001;2:17.
    • (2001) BMC Genet , vol.2 , pp. 17
    • Bennett, R.R.1    den Dunnen, J.2    O'Brien, K.F.3    Darras, B.T.4    Kunkel, L.M.5
  • 91
    • 0033583984 scopus 로고    scopus 로고
    • Signs and symptoms of Duchenne muscular dystrophy and Becker muscular dystrophy among carriers in The Netherlands: A cohort study
    • Hoogerwaard EM, Bakker E, Ippel PF, Oosterwijk JC, Majoor-Krakauer DF, Leschot NJ, et al. Signs and symptoms of Duchenne muscular dystrophy and Becker muscular dystrophy among carriers in The Netherlands: A cohort study. Lancet 1999;353:2116-9.
    • (1999) Lancet , vol.353 , pp. 2116-2119
    • Hoogerwaard, E.M.1    Bakker, E.2    Ippel, P.F.3    Oosterwijk, J.C.4    Majoor-Krakauer, D.F.5    Leschot, N.J.6
  • 92
    • 0024334813 scopus 로고
    • A survey of manifesting carriers of Duchenne and Becker muscular dystrophy in Wales
    • Norman A, Harper P. A survey of manifesting carriers of Duchenne and Becker muscular dystrophy in Wales. Clin Genet 1989;36:31-7.
    • (1989) Clin Genet , vol.36 , pp. 31-37
    • Norman, A.1    Harper, P.2
  • 95
    • 42549155295 scopus 로고    scopus 로고
    • Life expectancy and death from cardiomyopathy amongst carriers of Duchenne and Becker muscular dystrophy in Scotland
    • Holloway SM, Wilcox DE, Wilcox A, Dean JC, Berg JN, Goudie DR, et al. Life expectancy and death from cardiomyopathy amongst carriers of Duchenne and Becker muscular dystrophy in Scotland. Heart 2008;94:633-6.
    • (2008) Heart , vol.94 , pp. 633-636
    • Holloway, S.M.1    Wilcox, D.E.2    Wilcox, A.3    Dean, J.C.4    Berg, J.N.5    Goudie, D.R.6
  • 96
    • 0029971310 scopus 로고    scopus 로고
    • Development of cardiomyopathy in female carriers of Duchenne and Becker muscular dystrophies
    • Politano L, Nigro V, Nigro G, Petretta VR, Passamano L, Papparella S, et al. Development of cardiomyopathy in female carriers of Duchenne and Becker muscular dystrophies. JAMA 1996;275:1335-8.
    • (1996) JAMA , vol.275 , pp. 1335-1338
    • Politano, L.1    Nigro, V.2    Nigro, G.3    Petretta, V.R.4    Passamano, L.5    Papparella, S.6
  • 97
    • 0024537135 scopus 로고
    • Mosaic expression of dystrophin in symptomatic carriers of Duchenne's muscular dystrophy
    • Arahata K, Ishihara T, Kamakura K, Tsukahara T, Ishiura S, Baba C, et al. Mosaic expression of dystrophin in symptomatic carriers of Duchenne's muscular dystrophy. N Engl J Med 1989;320:138-42.
    • (1989) N Engl J Med , vol.320 , pp. 138-142
    • Arahata, K.1    Ishihara, T.2    Kamakura, K.3    Tsukahara, T.4    Ishiura, S.5    Baba, C.6
  • 98
    • 33645746833 scopus 로고    scopus 로고
    • Dystrophin analysis in carriers of Duchenne and Becker muscular dystrophy
    • Hoogerwaard EM, Ginjaar IB, Bakker E, de Visser M. Dystrophin analysis in carriers of Duchenne and Becker muscular dystrophy. Neurology 2005;65:1984-6.
    • (2005) Neurology , vol.65 , pp. 1984-1986
    • Hoogerwaard, E.M.1    Ginjaar, I.B.2    Bakker, E.3    de Visser, M.4
  • 99
    • 33644670773 scopus 로고    scopus 로고
    • Cardiovascular health supervision for individuals affected by Duchenne or Becker muscular dystrophy
    • American Academy of Pediatrics Section on C
    • American Academy of Pediatrics Section on C, Cardiac S. Cardiovascular health supervision for individuals affected by Duchenne or Becker muscular dystrophy. Pediatrics 2005;116:1569-73.
    • (2005) Pediatrics , vol.116 , pp. 1569-1573
    • Cardiac, S.1
  • 101
    • 20844460732 scopus 로고    scopus 로고
    • Therapy insight: Cardiovascular complications associated with muscular dystrophies
    • McNally EM, MacLeod H. Therapy insight: Cardiovascular complications associated with muscular dystrophies. Nat Clin Pract Cardiovasc Med 2005;2:301-8.
    • (2005) Nat Clin Pract Cardiovasc Med , vol.2 , pp. 301-308
    • McNally, E.M.1    MacLeod, H.2
  • 104
    • 26944453349 scopus 로고    scopus 로고
    • Randomized controlled trial of non-invasive ventilation (NIV) for nocturnal hypoventilation in neuromuscular and chest wall disease patients with daytime normocapnia
    • Ward S, Chatwin M, Heather S, Simonds AK. Randomized controlled trial of non-invasive ventilation (NIV) for nocturnal hypoventilation in neuromuscular and chest wall disease patients with daytime normocapnia. Thorax 2005;60:1019-24.
    • (2005) Thorax , vol.60 , pp. 1019-1024
    • Ward, S.1    Chatwin, M.2    Heather, S.3    Simonds, A.K.4
  • 105
    • 0036895043 scopus 로고    scopus 로고
    • Survival in Duchenne muscular dystrophy: Improvements in life expectancy since 1967 and the impact of home nocturnal ventilation
    • Eagle M, Baudouin SV, Chandler C, Giddings DR, Bullock R, Bushby K. Survival in Duchenne muscular dystrophy: Improvements in life expectancy since 1967 and the impact of home nocturnal ventilation. Neuromuscul Disord 2002;12:926-9.
    • (2002) Neuromuscul Disord , vol.12 , pp. 926-929
    • Eagle, M.1    Baudouin, S.V.2    Chandler, C.3    Giddings, D.R.4    Bullock, R.5    Bushby, K.6
  • 106
    • 34249099687 scopus 로고    scopus 로고
    • Managing Duchenne muscular dystrophy - the additive effect of spinal surgery and home nocturnal ventilation in improving survival
    • Eagle M, Bourke J, Bullock R, Gibson M, Mehta J, Giddings D, et al. Managing Duchenne muscular dystrophy - the additive effect of spinal surgery and home nocturnal ventilation in improving survival. Neuromuscul Disord 2007;17:470-5.
    • (2007) Neuromuscul Disord , vol.17 , pp. 470-475
    • Eagle, M.1    Bourke, J.2    Bullock, R.3    Gibson, M.4    Mehta, J.5    Giddings, D.6
  • 107
    • 27244431784 scopus 로고    scopus 로고
    • Effect of intermittent positive pressure ventilation on life-span and causes of death in Duchenne muscular dystrophy
    • Konagaya M, Sakai M, Wakayama T, Kimura S, Kuru S, Yasuma F. Effect of intermittent positive pressure ventilation on life-span and causes of death in Duchenne muscular dystrophy. Rinsho Shinkeigaku 2005;45:643-6.
    • (2005) Rinsho Shinkeigaku , vol.45 , pp. 643-646
    • Konagaya, M.1    Sakai, M.2    Wakayama, T.3    Kimura, S.4    Kuru, S.5    Yasuma, F.6
  • 108
    • 33751294514 scopus 로고    scopus 로고
    • Diurnal ventilation via mouthpiece: Survival in end-stage Duchenne patients
    • Toussaint M, Steens M, Wasteels G, Soudon P. Diurnal ventilation via mouthpiece: Survival in end-stage Duchenne patients. Eur Respir J 2006;28:549-55.
    • (2006) Eur Respir J , vol.28 , pp. 549-555
    • Toussaint, M.1    Steens, M.2    Wasteels, G.3    Soudon, P.4
  • 110
    • 34248201124 scopus 로고    scopus 로고
    • Orthopedic outcomes of long-term daily corticosteroid treatment in Duchenne muscular dystrophy
    • King WM, Ruttencutter R, Nagaraja HN, Matkovic V, Landoll J, Hoyle C, et al. Orthopedic outcomes of long-term daily corticosteroid treatment in Duchenne muscular dystrophy. Neurology 2007;68:1607-13.
    • (2007) Neurology , vol.68 , pp. 1607-1613
    • King, W.M.1    Ruttencutter, R.2    Nagaraja, H.N.3    Matkovic, V.4    Landoll, J.5    Hoyle, C.6
  • 111
    • 33847073275 scopus 로고    scopus 로고
    • Posterior spinal fusion for scoliosis in duchenne muscular dystrophy diminishes the rate of respiratory decline
    • Velasco MV, Colin AA, Zurakowski D, Darras BT, Shapiro F. Posterior spinal fusion for scoliosis in duchenne muscular dystrophy diminishes the rate of respiratory decline. Spine 2007;32:459-65.
    • (2007) Spine , vol.32 , pp. 459-465
    • Velasco, M.V.1    Colin, A.A.2    Zurakowski, D.3    Darras, B.T.4    Shapiro, F.5
  • 113
    • 15044353150 scopus 로고    scopus 로고
    • Taking steps towards reducing osteoporosis in Duchenne muscular dystrophy
    • Bachrach LK. Taking steps towards reducing osteoporosis in Duchenne muscular dystrophy. Neuromuscul Disord 2005;15:86-7.
    • (2005) Neuromuscul Disord , vol.15 , pp. 86-87
    • Bachrach, L.K.1
  • 114
    • 15044356774 scopus 로고    scopus 로고
    • Bone health in Duchenne muscular dystrophy: A workshop report from the meeting in Cincinnati, Ohio, July 8, 2004
    • Biggar WD, Bachrach LK, Henderson RC, Kalkwarf H, Plotkin H, Wong BL. Bone health in Duchenne muscular dystrophy: A workshop report from the meeting in Cincinnati, Ohio, July 8, 2004. Neuromuscul Disord 2005;15:80-5.
    • (2005) Neuromuscul Disord , vol.15 , pp. 80-85
    • Biggar, W.D.1    Bachrach, L.K.2    Henderson, R.C.3    Kalkwarf, H.4    Plotkin, H.5    Wong, B.L.6
  • 115
    • 15044360059 scopus 로고    scopus 로고
    • Quinlivan R, Roper H, Davie M, Shaw NJ, McDonagh J, Bushby K. Report of a Muscular Dystrophy Campaign funded workshop Birmingham, UK, January 16th 2004, Osteoporosis in Duchenne muscular dystrophy: Its prevalence, treatment and prevention. Neuromuscul Disord 2005;15:72-9.
    • Quinlivan R, Roper H, Davie M, Shaw NJ, McDonagh J, Bushby K. Report of a Muscular Dystrophy Campaign funded workshop Birmingham, UK, January 16th 2004, Osteoporosis in Duchenne muscular dystrophy: Its prevalence, treatment and prevention. Neuromuscul Disord 2005;15:72-9.
  • 116
    • 0036824144 scopus 로고    scopus 로고
    • Steroids in Duchenne muscular dystrophy: From clinical trials to genomic research
    • Muntoni F, Fisher I, Morgan JE, Abraham D. Steroids in Duchenne muscular dystrophy: From clinical trials to genomic research. Neuromuscul Disord 2002;12:S162-5.
    • (2002) Neuromuscul Disord , vol.12
    • Muntoni, F.1    Fisher, I.2    Morgan, J.E.3    Abraham, D.4
  • 118
    • 0025904393 scopus 로고
    • Mononuclear cell analysis of muscle biopsies in prednisone-treated and untreated Duchenne muscular dystrophy: CIDD Study Group
    • Kissel JT, Burrow KL, Rammohan KW, Mendell JR. Mononuclear cell analysis of muscle biopsies in prednisone-treated and untreated Duchenne muscular dystrophy: CIDD Study Group. Neurology 1991;41:667-72.
    • (1991) Neurology , vol.41 , pp. 667-672
    • Kissel, J.T.1    Burrow, K.L.2    Rammohan, K.W.3    Mendell, J.R.4
  • 120
    • 0031817490 scopus 로고    scopus 로고
    • Calcium influx inhibition by steroids and analogs in C2C12 skeletal muscle cells
    • Passaquin AC, Lhote P, Ruegg UT. Calcium influx inhibition by steroids and analogs in C2C12 skeletal muscle cells. Br J Pharmacol 1998;124:1751-9.
    • (1998) Br J Pharmacol , vol.124 , pp. 1751-1759
    • Passaquin, A.C.1    Lhote, P.2    Ruegg, U.T.3
  • 121
    • 0033779795 scopus 로고    scopus 로고
    • Deflazacort increases laminin expression and myogenic repair, and induces early persistent functional gain in mdx mouse muscular dystrophy
    • Anderson JE, Weber M, Vargas C. Deflazacort increases laminin expression and myogenic repair, and induces early persistent functional gain in mdx mouse muscular dystrophy. Cell Transplant 2000;9:551-64.
    • (2000) Cell Transplant , vol.9 , pp. 551-564
    • Anderson, J.E.1    Weber, M.2    Vargas, C.3
  • 122
    • 19944427852 scopus 로고    scopus 로고
    • Practice parameter: Corticosteroid treatment of Duchenne dystrophy: Report of the Quality Standards Subcommittee of the American Academy of Neurology and the Practice Committee of the Child Neurology Society
    • Moxley RT, 3rd, Ashwal S, Pandya S, Connolly A, Florence J, Mathews K, et al. Practice parameter: Corticosteroid treatment of Duchenne dystrophy: Report of the Quality Standards Subcommittee of the American Academy of Neurology and the Practice Committee of the Child Neurology Society. Neurology 2005;64:13-20.
    • (2005) Neurology , vol.64 , pp. 13-20
    • Moxley 3rd, R.T.1    Ashwal, S.2    Pandya, S.3    Connolly, A.4    Florence, J.5    Mathews, K.6
  • 123
    • 41749096186 scopus 로고    scopus 로고
    • Glucocorticoid corticosteroids for Duchenne muscular dystrophy.[update of Cochrane Database Syst Rev. 2004;(2):CD003725; PMID:15106215]
    • CD003725
    • Manzur AY, Kuntzer T, Pike M, Swan A. Glucocorticoid corticosteroids for Duchenne muscular dystrophy.[update of Cochrane Database Syst Rev. 2004;(2):CD003725; PMID:15106215]. Cochrane Database Syst Rev 2008:CD003725.
    • (2008) Cochrane Database Syst Rev
    • Manzur, A.Y.1    Kuntzer, T.2    Pike, M.3    Swan, A.4
  • 124
    • 4344588135 scopus 로고    scopus 로고
    • Report on the 124th ENMC International Workshop: Treatment of Duchenne muscular dystrophy: Defining the gold standards of management in the use of corticosteroids. 2-4 April 2004, Naarden, The Netherlands
    • Bushby K, Muntoni F, Urtizberea A, Hughes R, Griggs R. Report on the 124th ENMC International Workshop: Treatment of Duchenne muscular dystrophy: Defining the gold standards of management in the use of corticosteroids. 2-4 April 2004, Naarden, The Netherlands. Neuromuscul Disord 2004;14:526-34.
    • (2004) Neuromuscul Disord , vol.14 , pp. 526-534
    • Bushby, K.1    Muntoni, F.2    Urtizberea, A.3    Hughes, R.4    Griggs, R.5
  • 125
    • 0036018879 scopus 로고    scopus 로고
    • Corticosteroids in Duchenne muscular dystrophy: A reappraisal
    • Wong BL, Christopher C. Corticosteroids in Duchenne muscular dystrophy: A reappraisal. J Child Neurol 2002;17:183-90.
    • (2002) J Child Neurol , vol.17 , pp. 183-190
    • Wong, B.L.1    Christopher, C.2
  • 127
    • 0025869545 scopus 로고
    • Prednisone in Duchenne dystrophy: A randomized, controlled trial defining the time course and dose response
    • Griggs RC, Moxley RT 3rd, Mendell JR, Fenichel GM, Brooke MH, Pestronk A, et al. Prednisone in Duchenne dystrophy: A randomized, controlled trial defining the time course and dose response. Arch Neurol 1991;48:383-8.
    • (1991) Arch Neurol , vol.48 , pp. 383-388
    • Griggs, R.C.1    Moxley 3rd, R.T.2    Mendell, J.R.3    Fenichel, G.M.4    Brooke, M.H.5    Pestronk, A.6
  • 128
    • 0027499704 scopus 로고
    • Duchenne dystrophy: Randomized, controlled trial of prednisone (18 months) and azathioprine (12 months)
    • Griggs RC, Moxley RT 3rd, Mendell JR, Fenichel GM, Brooke MH, Pestronk A, et al. Duchenne dystrophy: Randomized, controlled trial of prednisone (18 months) and azathioprine (12 months). Neurology 1993;43:520-7.
    • (1993) Neurology , vol.43 , pp. 520-527
    • Griggs, R.C.1    Moxley 3rd, R.T.2    Mendell, J.R.3    Fenichel, G.M.4    Brooke, M.H.5    Pestronk, A.6
  • 129
    • 0025860096 scopus 로고
    • A comparison of daily and alternate-day prednisone therapy in the treatment of Duchenne muscular dystrophy
    • Fenichel GM, Mendell JR, Moxley RT 3rd, Griggs RC, Brooke MH, Miller JP, et al. A comparison of daily and alternate-day prednisone therapy in the treatment of Duchenne muscular dystrophy. Arch Neurol 1991;48:575-9.
    • (1991) Arch Neurol , vol.48 , pp. 575-579
    • Fenichel, G.M.1    Mendell, J.R.2    Moxley 3rd, R.T.3    Griggs, R.C.4    Brooke, M.H.5    Miller, J.P.6
  • 131
    • 0029011230 scopus 로고
    • Low-dose prednisolone treatment in Duchenne and Becker muscular dystrophy
    • Backman E, Henriksson KG. Low-dose prednisolone treatment in Duchenne and Becker muscular dystrophy. Neuromuscul Disord 1995;5:233-41.
    • (1995) Neuromuscul Disord , vol.5 , pp. 233-241
    • Backman, E.1    Henriksson, K.G.2
  • 132
    • 0015967746 scopus 로고
    • Failure of corticosteroid in the treatment of Duchenne (pseudo-hypertrophic) muscular dystrophy: Report of a clinically matched three year double-blind study
    • passim
    • Siegel IM, Miller JE, Ray RD. Failure of corticosteroid in the treatment of Duchenne (pseudo-hypertrophic) muscular dystrophy: Report of a clinically matched three year double-blind study. IMJ Ill Med J 1974;145:32-33 passim.
    • (1974) IMJ Ill Med J , vol.145 , pp. 32-33
    • Siegel, I.M.1    Miller, J.E.2    Ray, R.D.3
  • 135
    • 0036823601 scopus 로고    scopus 로고
    • An effective, low-dosage, intermittent schedule of prednisolone in the long-term treatment of early cases of Duchenne dystrophy
    • Kinali M, Mercuri E, Main M, Muntoni F, Dubowitz V. An effective, low-dosage, intermittent schedule of prednisolone in the long-term treatment of early cases of Duchenne dystrophy. Neuromuscul Disord 2002;12:S169-74.
    • (2002) Neuromuscul Disord , vol.12
    • Kinali, M.1    Mercuri, E.2    Main, M.3    Muntoni, F.4    Dubowitz, V.5
  • 136
    • 0025931256 scopus 로고
    • Prednisone in Duchenne dystrophy
    • Dubowitz V. Prednisone in Duchenne dystrophy. Neuromuscul Disord 1991;1:161-3.
    • (1991) Neuromuscul Disord , vol.1 , pp. 161-163
    • Dubowitz, V.1
  • 137
    • 0036893708 scopus 로고    scopus 로고
    • High dose weekly oral prednisone improves strength in boys with Duchenne muscular dystrophy
    • Connolly AM, Schierbecker J, Renna R, Florence J. High dose weekly oral prednisone improves strength in boys with Duchenne muscular dystrophy. Neuromuscul Disord 2002;12:917-25.
    • (2002) Neuromuscul Disord , vol.12 , pp. 917-925
    • Connolly, A.M.1    Schierbecker, J.2    Renna, R.3    Florence, J.4
  • 138
    • 0036042524 scopus 로고    scopus 로고
    • Remission of clinical signs in early duchenne muscular dystrophy on intermittent low-dosage prednisolone therapy
    • Dubowitz V, Kinali M, Main M, Mercuri E, Muntoni F. Remission of clinical signs in early duchenne muscular dystrophy on intermittent low-dosage prednisolone therapy. Eur J Paediatr Neurol 2002;6:153-9.
    • (2002) Eur J Paediatr Neurol , vol.6 , pp. 153-159
    • Dubowitz, V.1    Kinali, M.2    Main, M.3    Mercuri, E.4    Muntoni, F.5
  • 140
    • 0033812726 scopus 로고    scopus 로고
    • A multicenter, double-blind, randomized trial of deflazacort versus prednisone in Duchenne muscular dystrophy
    • Bonifati MD, Ruzza G, Bonometto P, Berardinelli A, Gorni K, Orcesi S, et al. A multicenter, double-blind, randomized trial of deflazacort versus prednisone in Duchenne muscular dystrophy. Muscle Nerve 2000;23:1344-7.
    • (2000) Muscle Nerve , vol.23 , pp. 1344-1347
    • Bonifati, M.D.1    Ruzza, G.2    Bonometto, P.3    Berardinelli, A.4    Gorni, K.5    Orcesi, S.6
  • 141
    • 0029555074 scopus 로고
    • Deflazacort vs. prednisone in Duchenne muscular dystrophy: Trends of an ongoing study
    • Reitter B. Deflazacort vs. prednisone in Duchenne muscular dystrophy: Trends of an ongoing study. Brain Dev 1995;17:39-43.
    • (1995) Brain Dev , vol.17 , pp. 39-43
    • Reitter, B.1
  • 142
    • 33646478253 scopus 로고    scopus 로고
    • Long-term benefits of deflazacort treatment for boys with Duchenne muscular dystrophy in their second decade
    • Biggar WD, Harris VA, Eliasoph L, Alman B. Long-term benefits of deflazacort treatment for boys with Duchenne muscular dystrophy in their second decade. Neuromuscul Disord 2006;16:249-55.
    • (2006) Neuromuscul Disord , vol.16 , pp. 249-255
    • Biggar, W.D.1    Harris, V.A.2    Eliasoph, L.3    Alman, B.4
  • 144
    • 27544464241 scopus 로고    scopus 로고
    • Corticosteroid treatment and functional improvement in Duchenne muscular dystrophy: Long-term effect
    • Balaban B, Matthews DJ, Clayton GH, Carry T. Corticosteroid treatment and functional improvement in Duchenne muscular dystrophy: Long-term effect. Am J Phys Med Rehabil 2005;84:843-50.
    • (2005) Am J Phys Med Rehabil , vol.84 , pp. 843-850
    • Balaban, B.1    Matthews, D.J.2    Clayton, G.H.3    Carry, T.4
  • 145
    • 4344633475 scopus 로고    scopus 로고
    • Effect of daily prednisone on independent ambulation in patients with Duchenne dystrophy treated for up to 15 years
    • Pandya S, Myers G, Moxley R. Effect of daily prednisone on independent ambulation in patients with Duchenne dystrophy treated for up to 15 years. Neuromuscul Disord 2001;11:630.
    • (2001) Neuromuscul Disord , vol.11 , pp. 630
    • Pandya, S.1    Myers, G.2    Moxley, R.3
  • 146
    • 33947182922 scopus 로고    scopus 로고
    • Effect of long-term steroids on cough efficiency and respiratory muscle strength in patients with Duchenne muscular dystrophy
    • Daftary AS, Crisanti M, Kalra M, Wong B, Amin R. Effect of long-term steroids on cough efficiency and respiratory muscle strength in patients with Duchenne muscular dystrophy. Pediatrics 2007;119:e320-4.
    • (2007) Pediatrics , vol.119
    • Daftary, A.S.1    Crisanti, M.2    Kalra, M.3    Wong, B.4    Amin, R.5
  • 149
    • 0037443583 scopus 로고    scopus 로고
    • Effects of deflazacort on left ventricular function in patients with Duchenne muscular dystrophy
    • Silversides CK, Webb GD, Harris VA, Biggar DW. Effects of deflazacort on left ventricular function in patients with Duchenne muscular dystrophy. Am J Cardiol 2003;91:769-72.
    • (2003) Am J Cardiol , vol.91 , pp. 769-772
    • Silversides, C.K.1    Webb, G.D.2    Harris, V.A.3    Biggar, D.W.4
  • 150
    • 1442331625 scopus 로고    scopus 로고
    • Steroid treatment and the development of scoliosis in males with duchenne muscular dystrophy
    • Alman BA, Raza SN, Biggar WD. Steroid treatment and the development of scoliosis in males with duchenne muscular dystrophy. J Bone Joint Surg Am 2004;86:519-24.
    • (2004) J Bone Joint Surg Am , vol.86 , pp. 519-524
    • Alman, B.A.1    Raza, S.N.2    Biggar, W.D.3
  • 152
    • 0035942323 scopus 로고    scopus 로고
    • A randomized efficacy and safety trial of oxandrolone in the treatment of Duchenne dystrophy
    • Fenichel GM, Griggs RC, Kissel J, Kramer TI, Mendell JR, Moxley RT, et al. A randomized efficacy and safety trial of oxandrolone in the treatment of Duchenne dystrophy. Neurology 2001;56:1075-9.
    • (2001) Neurology , vol.56 , pp. 1075-1079
    • Fenichel, G.M.1    Griggs, R.C.2    Kissel, J.3    Kramer, T.I.4    Mendell, J.R.5    Moxley, R.T.6
  • 153
    • 0027532390 scopus 로고
    • Cyclosporine increases muscular force generation in Duchenne muscular dystrophy
    • Sharma KR, Mynhier MA, Miller RG. Cyclosporine increases muscular force generation in Duchenne muscular dystrophy. Neurology 1993;43:527-32.
    • (1993) Neurology , vol.43 , pp. 527-532
    • Sharma, K.R.1    Mynhier, M.A.2    Miller, R.G.3
  • 154
    • 0343022262 scopus 로고    scopus 로고
    • Creatine monohydrate in muscular dystrophies: A double-blind, placebo-controlled clinical study
    • Walter MC, Lochmuller H, Reilich P, Klopstock T, Huber R, Hartard M, et al. Creatine monohydrate in muscular dystrophies: A double-blind, placebo-controlled clinical study. Neurology 2000;54:1848-50.
    • (2000) Neurology , vol.54 , pp. 1848-1850
    • Walter, M.C.1    Lochmuller, H.2    Reilich, P.3    Klopstock, T.4    Huber, R.5    Hartard, M.6
  • 156
  • 158
    • 0021130551 scopus 로고    scopus 로고
    • Mendell JR, Griggs RC, Moxley RT 3rd, Fenichel GM, Brooke MH, Miller JP, et al. Clinical investigation in Duchenne muscular dystrophy, IV: Double-blind controlled trial of leucine. Muscle Nerve 1984;7:535-541.
    • Mendell JR, Griggs RC, Moxley RT 3rd, Fenichel GM, Brooke MH, Miller JP, et al. Clinical investigation in Duchenne muscular dystrophy, IV: Double-blind controlled trial of leucine. Muscle Nerve 1984;7:535-541.
  • 160
    • 0020569886 scopus 로고
    • Clinical trials of vasoactive and antiserotonin drugs in Duchenne muscular dystrophy
    • Patten BM, Zeller RS. Clinical trials of vasoactive and antiserotonin drugs in Duchenne muscular dystrophy. Ann Clin Res 1983;15:164-6.
    • (1983) Ann Clin Res , vol.15 , pp. 164-166
    • Patten, B.M.1    Zeller, R.S.2
  • 161
    • 0023279816 scopus 로고
    • Double-blind study of the efficacy of an antiserotoninergic drug, pizotifen, in Duchenne's muscular dystrophy
    • Steru D, Paclet JP, Barthelet G, Gailliard G, Piton A, Monchartre E, et al. Double-blind study of the efficacy of an antiserotoninergic drug, pizotifen, in Duchenne's muscular dystrophy. Arch Fr Pediatr 1987;44:461-5.
    • (1987) Arch Fr Pediatr , vol.44 , pp. 461-465
    • Steru, D.1    Paclet, J.P.2    Barthelet, G.3    Gailliard, G.4    Piton, A.5    Monchartre, E.6
  • 162
    • 34548602367 scopus 로고    scopus 로고
    • Gene therapy for duchenne muscular dystrophy: Expectations and challenges
    • Rodino-Klapac LR, Chicoine LG, Kaspar BK, Mendell JR. Gene therapy for duchenne muscular dystrophy: Expectations and challenges. Arch Neurol 2007;64:1236-41.
    • (2007) Arch Neurol , vol.64 , pp. 1236-1241
    • Rodino-Klapac, L.R.1    Chicoine, L.G.2    Kaspar, B.K.3    Mendell, J.R.4
  • 163
    • 33745894322 scopus 로고    scopus 로고
    • rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice
    • Gregorevic P, Allen JM, Minami E, Blankinship MJ, Haraguchi M, Meuse L, et al. rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice. Nat Med 2006;12:787-9.
    • (2006) Nat Med , vol.12 , pp. 787-789
    • Gregorevic, P.1    Allen, J.M.2    Minami, E.3    Blankinship, M.J.4    Haraguchi, M.5    Meuse, L.6
  • 164
    • 0034610364 scopus 로고    scopus 로고
    • Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in MDX mouse model
    • Wang B, Li J, Xiao X. Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in MDX mouse model. Proc Natl Acad Sci USA 2000;97:13714-9.
    • (2000) Proc Natl Acad Sci USA , vol.97 , pp. 13714-13719
    • Wang, B.1    Li, J.2    Xiao, X.3
  • 165
    • 6344263893 scopus 로고    scopus 로고
    • Phase I study of dystrophin plasmid-based gene therapy in Duchenne/Becker muscular dystrophy
    • Romero NB, Braun S, Benveniste O, Leturcq F, Hogrel JY, Morris GE, et al. Phase I study of dystrophin plasmid-based gene therapy in Duchenne/Becker muscular dystrophy. Hum Gene Ther 2004;15:1065-76.
    • (2004) Hum Gene Ther , vol.15 , pp. 1065-1076
    • Romero, N.B.1    Braun, S.2    Benveniste, O.3    Leturcq, F.4    Hogrel, J.Y.5    Morris, G.E.6
  • 166
    • 33846419111 scopus 로고    scopus 로고
    • Non-viral gene therapy for Duchenne muscular dystrophy: Progress and challenges
    • Rando TA. Non-viral gene therapy for Duchenne muscular dystrophy: Progress and challenges. Biochim Biophys Acta 2007;1772:263-71.
    • (2007) Biochim Biophys Acta , vol.1772 , pp. 263-271
    • Rando, T.A.1
  • 167
    • 33645363853 scopus 로고    scopus 로고
    • Antisense oligonucleotides, exon skipping and the dystrophin gene transcript
    • Wilton SD, Fletcher S. Antisense oligonucleotides, exon skipping and the dystrophin gene transcript. Acta Myol 2005;24:222-9.
    • (2005) Acta Myol , vol.24 , pp. 222-229
    • Wilton, S.D.1    Fletcher, S.2
  • 168
    • 32244443828 scopus 로고    scopus 로고
    • Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology
    • Alter J, Lou F, Rabinowitz A, Yin H, Rosenfeld J, Wilton SD, et al. Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology. Nat Med 2006;12:175-7.
    • (2006) Nat Med , vol.12 , pp. 175-177
    • Alter, J.1    Lou, F.2    Rabinowitz, A.3    Yin, H.4    Rosenfeld, J.5    Wilton, S.D.6
  • 170
    • 38449095231 scopus 로고    scopus 로고
    • Clinical approaches in the treatment of Duchenne muscular dystrophy (DMD) using oligonucleotides
    • Bertoni C. Clinical approaches in the treatment of Duchenne muscular dystrophy (DMD) using oligonucleotides. Front Biosci 2008;13:517-27.
    • (2008) Front Biosci , vol.13 , pp. 517-527
    • Bertoni, C.1
  • 171
    • 34648854432 scopus 로고    scopus 로고
    • Genetic treatments in muscular dystrophies
    • Muntoni F, Wells D. Genetic treatments in muscular dystrophies. Curr Opin Neurol 2007;20:590-4.
    • (2007) Curr Opin Neurol , vol.20 , pp. 590-594
    • Muntoni, F.1    Wells, D.2
  • 173
    • 0043092426 scopus 로고    scopus 로고
    • Gentamicin administration in Duchenne patients with premature stop codon: Preliminary results
    • Politano L, Nigro G, Nigro V, Piluso G, Papparella S, Paciello O, et al. Gentamicin administration in Duchenne patients with premature stop codon: Preliminary results. Acta Myol 2003;22:15-21.
    • (2003) Acta Myol , vol.22 , pp. 15-21
    • Politano, L.1    Nigro, G.2    Nigro, V.3    Piluso, G.4    Papparella, S.5    Paciello, O.6
  • 175
    • 33751064254 scopus 로고    scopus 로고
    • Drug evaluation: PTC-124 - a potential treatment of cystic fibrosis and Duchenne muscular dystrophy
    • Hamed SA. Drug evaluation: PTC-124 - a potential treatment of cystic fibrosis and Duchenne muscular dystrophy. IDrugs 2006;9:783-9.
    • (2006) IDrugs , vol.9 , pp. 783-789
    • Hamed, S.A.1
  • 177
    • 33947529670 scopus 로고    scopus 로고
    • Safety, tolerability, and pharmacokinetics of PTC124: A nonaminoglycoside nonsense mutation suppressor, following single-and multiple-dose administration to healthy male and female adult volunteers
    • Hirawat S, Welch EM, Elfring GL, Northcutt VJ, Paushkin S, Hwang S, et al. Safety, tolerability, and pharmacokinetics of PTC124: A nonaminoglycoside nonsense mutation suppressor, following single-and multiple-dose administration to healthy male and female adult volunteers. J Clin Pharmacol 2007;47:430-44.
    • (2007) J Clin Pharmacol , vol.47 , pp. 430-444
    • Hirawat, S.1    Welch, E.M.2    Elfring, G.L.3    Northcutt, V.J.4    Paushkin, S.5    Hwang, S.6
  • 178
    • 56049124684 scopus 로고    scopus 로고
    • Phase 2 study of PTC124 for nonsense mutation suppression therapy of Duchenne Muscular Dystrophy (DMD)
    • Wong B, Bonnemann C, Finkel R, Flanigan KM, Sampson J, Sweeney L, et al. Phase 2 study of PTC124 for nonsense mutation suppression therapy of Duchenne Muscular Dystrophy (DMD). Ann Neurol 2007;62:S96.
    • (2007) Ann Neurol , vol.62
    • Wong, B.1    Bonnemann, C.2    Finkel, R.3    Flanigan, K.M.4    Sampson, J.5    Sweeney, L.6
  • 179
    • 0024540879 scopus 로고
    • Conversion of mdx myofibres from dystrophin-negative to -positive by injection of normal myoblasts
    • Partridge TA, Morgan JE, Coulton GR, Hoffman EP, Kunkel LM. Conversion of mdx myofibres from dystrophin-negative to -positive by injection of normal myoblasts. Nature 1989;337:176-9.
    • (1989) Nature , vol.337 , pp. 176-179
    • Partridge, T.A.1    Morgan, J.E.2    Coulton, G.R.3    Hoffman, E.P.4    Kunkel, L.M.5
  • 180
    • 0027166930 scopus 로고
    • Results of a triple blind clinical study of myoblast transplantations without immunosuppressive treatment in young boys with Duchenne muscular dystrophy
    • Tremblay JP, Malouin F, Roy R, Huard J, Bouchard JP, Satoh A, et al. Results of a triple blind clinical study of myoblast transplantations without immunosuppressive treatment in young boys with Duchenne muscular dystrophy. Cell Transplant 1993;2:99-112.
    • (1993) Cell Transplant , vol.2 , pp. 99-112
    • Tremblay, J.P.1    Malouin, F.2    Roy, R.3    Huard, J.4    Bouchard, J.P.5    Satoh, A.6
  • 181
    • 0028038998 scopus 로고
    • Human myoblast transplantation in immunodeficient and immunosuppressed mice: Evidence of rejection
    • Huard J, Roy R, Guerette B, Verreault S, Tremblay G, Tremblay JP. Human myoblast transplantation in immunodeficient and immunosuppressed mice: Evidence of rejection. Muscle Nerve 1994;17:224-34.
    • (1994) Muscle Nerve , vol.17 , pp. 224-234
    • Huard, J.1    Roy, R.2    Guerette, B.3    Verreault, S.4    Tremblay, G.5    Tremblay, J.P.6
  • 182
    • 0035831649 scopus 로고    scopus 로고
    • In vivo and in vitro correction of the mdx dystrophin gene nonsense mutation by short-fragment homologous replacement
    • Kapsa R, Quigley A, Lynch GS, Steeper K, Kornberg AJ, Gregorevic P, et al. In vivo and in vitro correction of the mdx dystrophin gene nonsense mutation by short-fragment homologous replacement. Hum Gene Ther 2001;12:629-42.
    • (2001) Hum Gene Ther , vol.12 , pp. 629-642
    • Kapsa, R.1    Quigley, A.2    Lynch, G.S.3    Steeper, K.4    Kornberg, A.J.5    Gregorevic, P.6
  • 183
    • 0036211418 scopus 로고    scopus 로고
    • Dystrophin gene repair in mdx muscle precursor cells in vitro and in vivo mediated by RNA-DNA chimeric oligonucleotides
    • Bertoni C, Rando TA. Dystrophin gene repair in mdx muscle precursor cells in vitro and in vivo mediated by RNA-DNA chimeric oligonucleotides. Hum Gene Ther 2002;13:707-18.
    • (2002) Hum Gene Ther , vol.13 , pp. 707-718
    • Bertoni, C.1    Rando, T.A.2
  • 184
    • 0033917878 scopus 로고    scopus 로고
    • Myogenic stem cells for the therapy of primary myopathies: Wishful thinking or therapeutic perspective?
    • Cossu G, Mavilio F. Myogenic stem cells for the therapy of primary myopathies: Wishful thinking or therapeutic perspective? J Clin Invest 2000;105:1669-74.
    • (2000) J Clin Invest , vol.105 , pp. 1669-1674
    • Cossu, G.1    Mavilio, F.2
  • 185
    • 0006462275 scopus 로고
    • Defective myoblasts identified in Duchenne muscular dystrophy
    • Blau HM, Webster C, Pavlath GK. Defective myoblasts identified in Duchenne muscular dystrophy. Proc Natl Acad Sci USA 1983;80:4856-60.
    • (1983) Proc Natl Acad Sci USA , vol.80 , pp. 4856-4860
    • Blau, H.M.1    Webster, C.2    Pavlath, G.K.3
  • 187
    • 34547099074 scopus 로고    scopus 로고
    • Evaluation of Sca-1 and c-Kit as selective markers for muscle remodelling by nonhemopoietic bone marrow cells
    • Wong SH, Lowes KN, Bertoncello I, Quigley AF, Simmons PJ, Cook MJ, et al. Evaluation of Sca-1 and c-Kit as selective markers for muscle remodelling by nonhemopoietic bone marrow cells. Stem Cells 2007;25:1364-74.
    • (2007) Stem Cells , vol.25 , pp. 1364-1374
    • Wong, S.H.1    Lowes, K.N.2    Bertoncello, I.3    Quigley, A.F.4    Simmons, P.J.5    Cook, M.J.6
  • 188
    • 0027255549 scopus 로고
    • Dystrophin-related protein, utrophin, in normal and dystrophic human fetal skeletal muscle
    • Clerk A, Morris GE, Dubowitz V, Davies KE, Sewry CA. Dystrophin-related protein, utrophin, in normal and dystrophic human fetal skeletal muscle. Histochem J 1993;25:554-61.
    • (1993) Histochem J , vol.25 , pp. 554-561
    • Clerk, A.1    Morris, G.E.2    Dubowitz, V.3    Davies, K.E.4    Sewry, C.A.5
  • 189
    • 33644975603 scopus 로고    scopus 로고
    • Utrophin upregulation for treating Duchenne or Becker muscular dystrophy: How close are we?
    • Miura P, Jasmin BJ. Utrophin upregulation for treating Duchenne or Becker muscular dystrophy: How close are we? Trends Mol Med 2006;12:122-9.
    • (2006) Trends Mol Med , vol.12 , pp. 122-129
    • Miura, P.1    Jasmin, B.J.2
  • 190
    • 0037306124 scopus 로고    scopus 로고
    • Cardiac assessment in childhood carriers of Duchenne and Becker muscular dystrophies
    • Nolan MA, Jones OD, Pedersen RL, Johnston HM. Cardiac assessment in childhood carriers of Duchenne and Becker muscular dystrophies. Neuromuscul Disord 2003;13:129-32.
    • (2003) Neuromuscul Disord , vol.13 , pp. 129-132
    • Nolan, M.A.1    Jones, O.D.2    Pedersen, R.L.3    Johnston, H.M.4


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