-
1
-
-
16444378040
-
Dystrophinopathies
-
Engel AG, Franzini-Armstrong C, editors 3rd ed. New York: McGraw-Hill
-
Engel AG, Ozawa E. Dystrophinopathies. In Engel AG, Franzini-Armstrong C, editors. Myology. 3rd ed. New York: McGraw-Hill; 2004. pp. 961-1025.
-
(2004)
Myology
-
-
Engel, A.G.1
Ozawa, E.2
-
2
-
-
0025869545
-
Prednisone in Duchenne dystrophy. A randomized, controlled trial defining the time course and dose response. Clinical Investigation of Duchenne Dystrophy Group
-
Griggs RC, Moxley RT III, Mendell JR, et al. Prednisone in Duchenne dystrophy. A randomized, controlled trial defining the time course and dose response. Clinical Investigation of Duchenne Dystrophy Group. Arch Neurol 1991; 48:383-388.
-
(1991)
Arch Neurol
, vol.48
, pp. 383-388
-
-
Griggs, R.C.1
Moxley Iii., R.T.2
Mendell, J.R.3
-
3
-
-
0033812726
-
A multicenter, double-blind, randomized trial of deflazacort versus prednisone in Duchenne muscular dystrophy
-
Bonifati MD, Ruzza G, Bonometto P, et al. A multicenter, double-blind, randomized trial of deflazacort versus prednisone in Duchenne muscular dystrophy. Muscle Nerve 2000; 23:1344-1347.
-
(2000)
Muscle Nerve
, vol.23
, pp. 1344-1347
-
-
Bonifati, M.D.1
Ruzza, G.2
Bonometto, P.3
-
4
-
-
78650186613
-
Use of corticosteroids in a populationbased cohort of boys with Duchenne and Becker muscular dystrophy
-
Matthews DJ, James KA, Miller LA, et al. Use of corticosteroids in a populationbased cohort of boys with Duchenne and Becker muscular dystrophy. J Child Neurol 2010; 25:1319-1324.
-
(2010)
J Child Neurol
, vol.25
, pp. 1319-1324
-
-
Matthews, D.J.1
James, K.A.2
Miller, L.A.3
-
5
-
-
80051500586
-
Randomized, blinded trial of weekend vs. daily prednisone in Duchenne muscular dystrophy
-
Escolar DM, Hache LP, Clemens PR, et al. Randomized, blinded trial of weekend vs. daily prednisone in Duchenne muscular dystrophy. Neurology 2011; 77:444-452.
-
(2011)
Neurology
, vol.77
, pp. 444-452
-
-
Escolar, D.M.1
Hache, L.P.2
Clemens, P.R.3
-
6
-
-
79959297748
-
Cardiomyopathy of Duchenne muscular dystrophy: Current understanding and future directions
-
Spurney CF. Cardiomyopathy of Duchenne muscular dystrophy: current understanding and future directions. Muscle Nerve 2011; 44:8-19.
-
(2011)
Muscle Nerve
, vol.44
, pp. 8-19
-
-
Spurney, C.F.1
-
7
-
-
79951479823
-
Losartan decreases cardiac muscle fibrosis and improves cardiac function in dystrophin-deficient mdx mice
-
Spurney CF, Sali A, Guerron AD, et al. Losartan decreases cardiac muscle fibrosis and improves cardiac function in dystrophin-deficient mdx mice. J Cardiovasc Pharmacol Ther 2011; 16:87-95.
-
(2011)
J Cardiovasc Pharmacol Ther
, vol.16
, pp. 87-95
-
-
Spurney, C.F.1
Sali, A.2
Guerron, A.D.3
-
8
-
-
84857668964
-
Effects of steroids and angiotensin converting enzyme inhibition on circumferential strain in boys with Duchenne muscular dystrophy: A cross-sectional and longitudinal study utilizing cardiovascular magnetic resonance
-
Hor KN, Mazur W, Taylor MD, et al. Effects of steroids and angiotensin converting enzyme inhibition on circumferential strain in boys with Duchenne muscular dystrophy: a cross-sectional and longitudinal study utilizing cardiovascular magnetic resonance. J Cardiovasc Magn Reson 2011; 13:60.
-
(2011)
J Cardiovasc Magn Reson
, vol.13
, pp. 60
-
-
Hor, K.N.1
Mazur, W.2
Taylor, M.D.3
-
9
-
-
84858338127
-
Nitric oxide donor and nonsteroidal anti-inflammatory drugs as a therapy for muscular dystrophies: Evidence from a safety study with pilot efficacy measures in adult dystrophic patients
-
D'Angelo MG, Gandossini S, Martinelli BF, et al. Nitric oxide donor and nonsteroidal anti-inflammatory drugs as a therapy for muscular dystrophies: evidence from a safety study with pilot efficacy measures in adult dystrophic patients. Pharmacol Res 2012; 65:472-479.
-
(2012)
Pharmacol Res
, vol.65
, pp. 472-479
-
-
D'Angelo, M.G.1
Gandossini, S.2
Martinelli, B.F.3
-
10
-
-
0025932274
-
Immunolocalization and developmental expression of dystrophin related protein in skeletal muscle
-
Khurana TS, Watkins SC, Chafey P, et al. Immunolocalization and developmental expression of dystrophin related protein in skeletal muscle. Neuromuscul Disord 1991; 1:185-194.
-
(1991)
Neuromuscul Disord
, vol.1
, pp. 185-194
-
-
Khurana, T.S.1
Watkins, S.C.2
Chafey, P.3
-
11
-
-
0033587139
-
Adenovirus-mediated utrophin gene transfer mitigates the dystrophic phenotype of mdx mouse muscles
-
DOI 10.1089/10430349950017987
-
Gilbert R, Nalbantoglu J, Petrof BJ, et al. Adenovirus-mediated utrophin gene transfer mitigates the dystrophic phenotype of mdx mouse muscles. Human Gene Ther 1999; 10:1299-1310. (Pubitemid 29267585)
-
(1999)
Human Gene Therapy
, vol.10
, Issue.8
, pp. 1299-1310
-
-
Gilbert, R.1
Nalbantoglu, J.2
Petrof, B.J.3
Ebihara, S.4
Guibinga, G.-H.5
Tinsley, J.M.6
Kamen, A.7
Massie, B.8
Davies, K.E.9
Karpati, G.10
-
12
-
-
0031727771
-
Expression of full-length utrophin prevents muscular dystrophy in mdx mice
-
DOI 10.1038/4033
-
Tinsley J, Deconinck N, Fisher R, et al. Expression of full-length utrophin prevents muscular dystrophy in mdx mice. Nature Med 1998; 4:1441-1444. (Pubitemid 28553471)
-
(1998)
Nature Medicine
, vol.4
, Issue.12
, pp. 1441-1444
-
-
Tinsley, J.1
Deconinck, N.2
Fisher, R.3
Kahn, D.4
Phelps, S.5
Gillis, J.-M.6
Davies, K.7
-
13
-
-
79551667507
-
Biglycan recruits utrophin to the sarcolemma and counters dystrophic pathology in mdx mice
-
Amenta AR, Yilmaz A, Bogdanovich S, et al. Biglycan recruits utrophin to the sarcolemma and counters dystrophic pathology in mdx mice. Proc Natl Acad Sci USA 2011; 108:762-767.
-
(2011)
Proc Natl Acad Sci USA
, vol.108
, pp. 762-767
-
-
Amenta, A.R.1
Yilmaz, A.2
Bogdanovich, S.3
-
14
-
-
79955856839
-
Discovery of 2-arylbenzoxazoles as upregulators of utrophin production for the treatment of Duchenne muscular dystrophy
-
Chancellor DR, Davies KE, De MO, et al. Discovery of 2-arylbenzoxazoles as upregulators of utrophin production for the treatment of Duchenne muscular dystrophy. J Med Chem 2011; 54:3241-3250.
-
(2011)
J Med Chem
, vol.54
, pp. 3241-3250
-
-
Chancellor, D.R.1
Davies, K.E.2
De, M.O.3
-
15
-
-
79960918733
-
Discovery and SAR of 2-arylbenzotriazoles and 2-arylindazoles as potential treatments for Duchenne muscular dystrophy
-
De MO, Dorgan CR, Johnson PD, et al. Discovery and SAR of 2-arylbenzotriazoles and 2-arylindazoles as potential treatments for Duchenne muscular dystrophy. Bioorg Med Chem Lett 2011; 21:4828-4831.
-
(2011)
Bioorg Med Chem Lett
, vol.21
, pp. 4828-4831
-
-
De Mo Dorgan, C.R.1
Johnson, P.D.2
-
16
-
-
79955867741
-
Daily treatment with SMTC1100, a novel small molecule utrophin upregulator, dramatically reduces the dystrophic symptoms in the mdx mouse
-
Tinsley JM, Fairclough RJ, Storer R, et al. Daily treatment with SMTC1100, a novel small molecule utrophin upregulator, dramatically reduces the dystrophic symptoms in the mdx mouse. PLoS One 2011; 6:e19189.
-
(2011)
PLoS One
, vol.6
-
-
Tinsley, J.M.1
Fairclough, R.J.2
Storer, R.3
-
17
-
-
80054779433
-
Drug discovery for Duchenne muscular dystrophy via utrophin promoter activation screening
-
Moorwood C, Lozynska O, Suri N, et al. Drug discovery for Duchenne muscular dystrophy via utrophin promoter activation screening. PLoS One 2011; 6:e26169.
-
(2011)
PLoS One
, vol.6
-
-
Moorwood, C.1
Lozynska, O.2
Suri, N.3
-
18
-
-
84455172952
-
Translational regulation of utrophin by miRNAs
-
Basu U, Lozynska O, Moorwood C, et al. Translational regulation of utrophin by miRNAs. PLoS One 2011; 6:e29376.
-
(2011)
PLoS One
, vol.6
-
-
Basu, U.1
Lozynska, O.2
Moorwood, C.3
-
19
-
-
33746766278
-
Entries in the Leiden Duchenne muscular dystrophy mutation database: An overview of mutation types and paradoxical cases that confirm the reading-frame rule
-
DOI 10.1002/mus.20586
-
artsma-Rus A, van Deutekom JC, Fokkema IF, et al. 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; 34:135-144. (Pubitemid 44167796)
-
(2006)
Muscle and Nerve
, vol.34
, Issue.2
, pp. 135-144
-
-
Aartsma-Rus, A.1
Van Deutekom, J.C.T.2
Fokkema, I.F.3
Van Ommen, G.-J.B.4
Den Dunnen, J.T.5
-
20
-
-
77952938084
-
Gentamicin-induced readthrough of stop codons inDuchennemuscular dystrophy
-
Malik V, Rodino-Klapac LR, Viollet L, et al. Gentamicin-induced readthrough of stop codons inDuchennemuscular dystrophy.AnnNeurol2010; 67:771-780.
-
(2010)
AnnNeurol
, vol.67
, pp. 771-780
-
-
Malik, V.1
Rodino-Klapac, L.R.2
Viollet, L.3
-
21
-
-
0043092426
-
Gentamicin administration in Duchenne patients with premature stop codon. Preliminary results
-
Politano L, Nigro G, Nigro V, et al. Gentamicin administration in Duchenne patients with premature stop codon. Preliminary results. Acta Myol 2003; 22:15-21. (Pubitemid 36966404)
-
(2003)
Acta Myologica
, vol.22
, Issue.MAY
, pp. 15-21
-
-
Politano, L.1
Nigro, G.2
Nigro, V.3
Pilus, G.4
Papparella, S.5
Paciello, O.6
Comi, L.I.7
-
22
-
-
0034982292
-
Gentamicin treatment of Duchenne and Becker muscular dystrophy due to nonsense mutations
-
DOI 10.1002/ana.1023
-
Wagner KR, Hamed S, Hadley DW, et al. Gentamicin treatment of Duchenne and Becker muscular dystrophy due to nonsense mutations. Ann Neurol 2001; 49:706-711. (Pubitemid 32530236)
-
(2001)
Annals of Neurology
, vol.49
, Issue.6
, pp. 706-711
-
-
Wagner, K.R.1
Hamed, S.2
Hadley, D.W.3
Gropman, A.L.4
Burstein, A.H.5
Escolar, D.M.6
Hoffman, E.P.7
Fischbeck, K.H.8
-
23
-
-
77956311645
-
Read-through strategies for suppression of nonsense mutations in Duchenne/Becker muscular dystrophy: Aminoglycosides and ataluren (PTC124)
-
Finkel RS. Read-through strategies for suppression of nonsense mutations in Duchenne/Becker muscular dystrophy: aminoglycosides and ataluren (PTC124). J Child Neurol 2010; 25:1158-1164.
-
(2010)
J Child Neurol
, vol.25
, pp. 1158-1164
-
-
Finkel, R.S.1
-
24
-
-
0036773266
-
Fracture prevalence in Duchenne muscular dystrophy
-
DOI 10.1017/S0012162201002778
-
McDonald DG, Kinali M, Gallagher AC, et al. Fracture prevalence in Duchenne muscular dystrophy. Dev Med Child Neurol 2002; 44:695-698. (Pubitemid 35230031)
-
(2002)
Developmental Medicine and Child Neurology
, vol.44
, Issue.10
, pp. 695-698
-
-
McDonald, D.G.M.1
Kinali, M.2
Gallagher, A.C.3
Mercuri, E.4
Muntoni, F.5
Roper, H.6
Jardine, P.7
Jones, D.H.8
Pike, M.G.9
-
26
-
-
0017646399
-
Fractures of long bones in duchenne muscular dystrophy
-
Siegel IM. Fractures of long bones in Duchenne muscular dystrophy. J Trauma 1977; 17:219-222. (Pubitemid 8096954)
-
(1977)
Journal of Trauma
, vol.17
, Issue.3
, pp. 219-222
-
-
Siegel, I.M.1
-
27
-
-
0034922496
-
Fracture risk in patients with muscular dystrophy and spinal muscular atrophy
-
Vestergaard P, Glerup H, Steffensen BF, et al. Fracture risk in patients with muscular dystrophy and spinal muscular atrophy. J Rehabil Med 2001; 33:150-155. (Pubitemid 32700475)
-
(2001)
Journal of Rehabilitation Medicine
, vol.33
, Issue.4
, pp. 150-155
-
-
Vestergaard, P.1
Glerup, H.2
Steffensen, B.F.3
Rejnmark, L.4
Rahbek, J.5
Mosekilde, L.6
-
28
-
-
72149108443
-
Diagnosis and management of Duchenne muscular dystrophy, part 1: Diagnosis, and pharmacological and psychosocial management
-
Bushby K, Finkel R, Birnkrant DJ, et al. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management. Lancet Neurol 2010; 9:77-93.
-
(2010)
Lancet Neurol
, vol.9
, pp. 77-93
-
-
Bushby, K.1
Finkel, R.2
Birnkrant, D.J.3
-
29
-
-
76549130473
-
Diagnosis and management of Duchenne muscular dystrophy, part 2: Implementation of multidisciplinary care
-
Bushby K, Finkel R, Birnkrant DJ, et al. Diagnosis and management of Duchenne muscular dystrophy, part 2: implementation of multidisciplinary care. Lancet Neurol 2010; 9:177-189.
-
(2010)
Lancet Neurol
, vol.9
, pp. 177-189
-
-
Bushby, K.1
Finkel, R.2
Birnkrant, D.J.3
-
30
-
-
0041846566
-
Vertebral fractures in boys with Duchenne muscular dystrophy
-
Bothwell JE, Gordon KE, Dooley JM, et al. Vertebral fractures in boys with Duchenne muscular dystrophy. Clin Pediatr (Phila) 2003; 42:353-356. (Pubitemid 36909149)
-
(2003)
Clinical Pediatrics
, vol.42
, Issue.4
, pp. 353-356
-
-
Bothwell, J.E.1
Gordon, K.E.2
Dooley, J.M.3
MacSween, J.4
Cummings, E.A.5
Salisbury, S.6
-
31
-
-
0036166717
-
Vertebral compression in Duchenne muscular dystrophy following deflazacort
-
DOI 10.1016/S0960-8966(01)00307-8, PII S0960896601003078
-
Talim B, Malaguti C, Gnudi S, et al. Vertebral compression in Duchenne muscular dystrophy following deflazacort. Neuromuscul Disord 2002; 12:294-295. (Pubitemid 34136914)
-
(2002)
Neuromuscular Disorders
, vol.12
, Issue.3
, pp. 294-295
-
-
Talim, B.1
Malaguti, C.2
Gnudi, S.3
Politano, L.4
Merlini, L.5
-
32
-
-
84861705331
-
Bisphosphonates for osteoporosis: Where do we go from here?
-
Whitaker M, Guo J, Kehoe T, Benson G. Bisphosphonates for osteoporosis: where do we go from here? N Engl J Med 2012; 366:2048-2051.
-
(2012)
N Engl J Med
, vol.366
, pp. 2048-2051
-
-
Whitaker, M.1
Guo, J.2
Kehoe, T.3
Benson, G.4
-
33
-
-
84868208578
-
The use of intravenous bisphosphonate therapy to treat vertebral fractures due to osteoporosis among boys with Duchenne muscular dystrophy
-
[Epub ahead of print]
-
Sbrocchi AM, Rauch F, Jacob P, et al. The use of intravenous bisphosphonate therapy to treat vertebral fractures due to osteoporosis among boys with Duchenne muscular dystrophy. Osteoporos Int 2012. [Epub ahead of print]
-
(2012)
Osteoporos Int
-
-
Sbrocchi, A.M.1
Rauch, F.2
Jacob, P.3
-
34
-
-
79551545969
-
Impact of bisphosphonates on survival for patients with Duchenne muscular dystrophy
-
Gordon KE, Dooley JM, Sheppard KM, et al. Impact of bisphosphonates on survival for patients with Duchenne muscular dystrophy. Pediatrics 2011; 127:e353-e358.
-
(2011)
Pediatrics
, vol.127
-
-
Gordon, K.E.1
Dooley, J.M.2
Sheppard, K.M.3
-
35
-
-
13444259605
-
Alendronate in the treatment of low bone mass in steroid-treated boys with Duchenne's muscular dystrophy
-
DOI 10.1016/j.apmr.2004.04.021
-
Hawker GA, Ridout R, Harris VA, et al. Alendronate in the treatment of low bone mass in steroid-treated boys with Duchennes muscular dystrophy. Arch Phys Med Rehabil 2005; 86:284-288. (Pubitemid 40215291)
-
(2005)
Archives of Physical Medicine and Rehabilitation
, vol.86
, Issue.2
, pp. 284-288
-
-
Hawker, G.A.1
Ridout, R.2
Harris, V.A.3
Chase, C.C.4
Fielding, L.J.5
Biggar, W.D.6
-
36
-
-
33748854083
-
Myotonic dystrophy
-
Engel AG, Franzini-Armstrong C, editors 3rd ed. New York: McGraw-Hill
-
Harper PS, Monckton DG. Myotonic dystrophy. In Engel AG, Franzini-Armstrong C, editors. Myology. 3rd ed. New York: McGraw-Hill; 2004. pp. 1039-1076.
-
(2004)
Myology
, pp. 1039-1076
-
-
Harper, P.S.1
Monckton, D.G.2
-
37
-
-
84859521683
-
Myotonic dystrophy type 1 or Steinert's disease
-
Romeo V. Myotonic dystrophy type 1 or Steinert's disease. Adv Exp Med Biol 2012; 724:239-257.
-
(2012)
Adv Exp Med Biol
, vol.724
, pp. 239-257
-
-
Romeo, V.1
-
38
-
-
77951848201
-
Mexiletine is an effective antimyotonia treatment in myotonic dystrophy type 1
-
Logigian EL, Martens WB, Moxley RT, et al. Mexiletine is an effective antimyotonia treatment in myotonic dystrophy type 1. Neurology 2010; 74:1441-1448.
-
(2010)
Neurology
, vol.74
, pp. 1441-1448
-
-
Logigian, E.L.1
Martens, W.B.2
Moxley, R.T.3
-
39
-
-
33644510962
-
Facioscapulohumeral muscular dystrophy and scapuloperoneal disorders
-
Engel AG, Franzini-Armstrong C, editors 3rd ed. New York: McGraw-Hill
-
Flanigan KM. Facioscapulohumeral muscular dystrophy and scapuloperoneal disorders. In Engel AG, Franzini-Armstrong C, editors. Myology. 3rd ed. New York: McGraw-Hill; 2004. pp. 1123-1133.
-
(2004)
Myology
, pp. 1123-1133
-
-
Flanigan, K.M.1
-
40
-
-
79957512849
-
The muscular dystrophies: Distinct pathogenic mechanisms invite novel therapeutic approaches
-
Sahenk Z, Mendell Jr. The muscular dystrophies: distinct pathogenic mechanisms invite novel therapeutic approaches. Curr Rheumatol Rep 2011; 13:199-207.
-
(2011)
Curr Rheumatol Rep
, vol.13
, pp. 199-207
-
-
Sahenk, Z.1
Mendell, J.R.2
-
41
-
-
84856255633
-
Changes in pain-related beliefs, coping, and catastrophizing predict changes in pain intensity, pain interference, and psychological functioning in individuals with myotonic muscular dystrophy and facioscapulohumeral dystrophy
-
Nieto R, Raichle KA, Jensen MP, Miro J. Changes in pain-related beliefs, coping, and catastrophizing predict changes in pain intensity, pain interference, and psychological functioning in individuals with myotonic muscular dystrophy and facioscapulohumeral dystrophy. Clin J Pain 2012; 28:47-54.
-
(2012)
Clin J Pain
, vol.28
, pp. 47-54
-
-
Nieto, R.1
Raichle, K.A.2
Jensen, M.P.3
Miro, J.4
|