-
1
-
-
85018858970
-
The burden, epidemiology, costs and treatment for Duchenne muscular dystrophy: an evidence review
-
Ryder, S, Leadley, RM, Armstrong, N, et al. The burden, epidemiology, costs and treatment for Duchenne muscular dystrophy: an evidence review. Orphanet J Rare Dis, 12, 2017, 79.
-
(2017)
Orphanet J Rare Dis
, vol.12
, pp. 79
-
-
Ryder, S.1
Leadley, R.M.2
Armstrong, N.3
-
2
-
-
84859181514
-
Evidence-based path to newborn screening for Duchenne muscular dystrophy
-
Mendell, JR, Shilling, C, Leslie, ND, et al. Evidence-based path to newborn screening for Duchenne muscular dystrophy. Ann Neurol 71 (2012), 304–313.
-
(2012)
Ann Neurol
, vol.71
, pp. 304-313
-
-
Mendell, J.R.1
Shilling, C.2
Leslie, N.D.3
-
3
-
-
84884588696
-
Newborn bloodspot screening for Duchenne muscular dystrophy: 21 years experience in Wales (UK)
-
Moat, SJ, Bradley, DM, Salmon, R, Clarke, A, Hartley, L, Newborn bloodspot screening for Duchenne muscular dystrophy: 21 years experience in Wales (UK). Eur J Hum Genet 21 (2013), 1049–1053.
-
(2013)
Eur J Hum Genet
, vol.21
, pp. 1049-1053
-
-
Moat, S.J.1
Bradley, D.M.2
Salmon, R.3
Clarke, A.4
Hartley, L.5
-
4
-
-
84949313313
-
Advances in genetic therapeutic strategies for Duchenne muscular dystrophy
-
Guiraud, S, Chen, H, Burns, DT, Davies, KE, Advances in genetic therapeutic strategies for Duchenne muscular dystrophy. Exp Physiol 100 (2015), 1458–1467.
-
(2015)
Exp Physiol
, vol.100
, pp. 1458-1467
-
-
Guiraud, S.1
Chen, H.2
Burns, D.T.3
Davies, K.E.4
-
5
-
-
85042015678
-
State of the art advances in Duchenne muscular dystrophy
-
Van Ruiten, H, Bushby, K, Guglieri, M, State of the art advances in Duchenne muscular dystrophy. EMJ 2 (2017), 90–99.
-
(2017)
EMJ
, vol.2
, pp. 90-99
-
-
Van Ruiten, H.1
Bushby, K.2
Guglieri, M.3
-
6
-
-
72149108443
-
Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management
-
Bushby, K, Finkel, R, Birnkrant, DJ, et al., for the DMD Care Considerations Working Group. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management. Lancet Neurol 9 (2010), 77–93.
-
(2010)
Lancet Neurol
, vol.9
, pp. 77-93
-
-
Bushby, K.1
Finkel, R.2
Birnkrant, D.J.3
-
7
-
-
76549130473
-
Diagnosis and management of Duchenne muscular dystrophy, part 2: implementation of multidisciplinary care
-
Bushby, K, Finkel, R, Birnkrant, DJ, et al., for the DMD Care Considerations Working Group. Diagnosis and management of Duchenne muscular dystrophy, part 2: implementation of multidisciplinary care. Lancet Neurol 9 (2010), 177–189.
-
(2010)
Lancet Neurol
, vol.9
, pp. 177-189
-
-
Bushby, K.1
Finkel, R.2
Birnkrant, D.J.3
-
8
-
-
85042010171
-
Family guide in different languages
-
(accessed March 8, 2016).
-
TREAT-NMD. Family guide in different languages. http://www.treat-nmd.eu/dmd/care/family-guide/translations/, 2016 (accessed March 8, 2016).
-
(2016)
-
-
-
9
-
-
84929378093
-
Contemporary cardiac issues in Duchenne muscular dystrophy. Working Group of the National Heart, Lung, and Blood Institute in collaboration with Parent Project Muscular Dystrophy
-
McNally, EM, Kaltman, JR, Benson, DW, et al. the Working Group of the National Heart, Lung, and Blood Institute, Parent Project Muscular Dystrophy. Contemporary cardiac issues in Duchenne muscular dystrophy. Working Group of the National Heart, Lung, and Blood Institute in collaboration with Parent Project Muscular Dystrophy. Circulation 131 (2015), 1590–1598.
-
(2015)
Circulation
, vol.131
, pp. 1590-1598
-
-
McNally, E.M.1
Kaltman, J.R.2
Benson, D.W.3
-
10
-
-
77956104812
-
The respiratory management of patients with Duchenne muscular dystrophy: a DMD care considerations working group specialty article
-
Birnkrant, DJ, Bushby, KM, Amin, RS, et al. The respiratory management of patients with Duchenne muscular dystrophy: a DMD care considerations working group specialty article. Pediatr Pulmonol 45 (2010), 739–748.
-
(2010)
Pediatr Pulmonol
, vol.45
, pp. 739-748
-
-
Birnkrant, D.J.1
Bushby, K.M.2
Amin, R.S.3
-
11
-
-
84869160655
-
Improvement of survival in Duchenne muscular dystrophy: retrospective analysis of 835 patients
-
Passamano, L, Taglia, A, Palladino, A, et al. Improvement of survival in Duchenne muscular dystrophy: retrospective analysis of 835 patients. Acta Myol 31 (2012), 121–125.
-
(2012)
Acta Myol
, vol.31
, pp. 121-125
-
-
Passamano, L.1
Taglia, A.2
Palladino, A.3
-
12
-
-
85008440540
-
Study of Duchenne muscular dystrophy long-term survivors aged 40 years and older living in specialized institutions in Japan
-
Saito, T, Kawai, M, Kimura, E, et al. Study of Duchenne muscular dystrophy long-term survivors aged 40 years and older living in specialized institutions in Japan. Neuromuscul Disord 27 (2017), 107–114.
-
(2017)
Neuromuscul Disord
, vol.27
, pp. 107-114
-
-
Saito, T.1
Kawai, M.2
Kimura, E.3
-
13
-
-
84976354813
-
Recent advances in innovative therapeutic approaches for Duchenne muscular dystrophy: from discovery to clinical trials
-
Shimizu-Motohashi, Y, Miyatake, S, Komaki, H, Takeda, S, Aoki, Y, Recent advances in innovative therapeutic approaches for Duchenne muscular dystrophy: from discovery to clinical trials. Am J Transl Res 8 (2016), 2471–2489.
-
(2016)
Am J Transl Res
, vol.8
, pp. 2471-2489
-
-
Shimizu-Motohashi, Y.1
Miyatake, S.2
Komaki, H.3
Takeda, S.4
Aoki, Y.5
-
14
-
-
84995592213
-
Efficacy and safety of deflazacort vs prednisone and placebo for Duchenne muscular dystrophy
-
Griggs, RC, Miller, JP, Greenberg, CR, et al. Efficacy and safety of deflazacort vs prednisone and placebo for Duchenne muscular dystrophy. Neurology 87 (2016), 2123–2131.
-
(2016)
Neurology
, vol.87
, pp. 2123-2131
-
-
Griggs, R.C.1
Miller, J.P.2
Greenberg, C.R.3
-
15
-
-
85018333312
-
Developing standardized corticosteroid treatment for Duchenne muscular dystrophy
-
Guglieri, M, Bushby, K, McDermott, MP, et al. Developing standardized corticosteroid treatment for Duchenne muscular dystrophy. Contemp Clin Trials 58 (2017), 34–39.
-
(2017)
Contemp Clin Trials
, vol.58
, pp. 34-39
-
-
Guglieri, M.1
Bushby, K.2
McDermott, M.P.3
-
16
-
-
84957598991
-
Practice guideline update summary: Corticosteroid treatment of Duchenne muscular dystrophy: Report of the Guideline Development Subcommittee of the American Academy of Neurology
-
Gloss, D, Moxley, RT 3rd, Ashwal, S, Oskoui, M, Practice guideline update summary: Corticosteroid treatment of Duchenne muscular dystrophy: Report of the Guideline Development Subcommittee of the American Academy of Neurology. Neurology 86 (2016), 465–472.
-
(2016)
Neurology
, vol.86
, pp. 465-472
-
-
Gloss, D.1
Moxley, R.T.2
Ashwal, S.3
Oskoui, M.4
-
17
-
-
0003423053
-
The RAND/UCLA Appropriateness Method user's manual
-
RAND Santa Monica, CA
-
Fitch, K, Bernstein, SJ, Aguilar, MD, et al. The RAND/UCLA Appropriateness Method user's manual. 2001, RAND, Santa Monica, CA.
-
(2001)
-
-
Fitch, K.1
Bernstein, S.J.2
Aguilar, M.D.3
-
18
-
-
69249230932
-
Delayed diagnosis in Duchenne muscular dystrophy: data from the Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet)
-
Ciafaloni, E, Fox, DJ, Pandya, S, et al. Delayed diagnosis in Duchenne muscular dystrophy: data from the Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet). J Pediatr 155 (2009), 380–385.
-
(2009)
J Pediatr
, vol.155
, pp. 380-385
-
-
Ciafaloni, E.1
Fox, D.J.2
Pandya, S.3
-
19
-
-
84908334398
-
Motor and cognitive delay in Duchenne muscular dystrophy: implication for early diagnosis
-
Mirski, KT, Crawford, TO, Motor and cognitive delay in Duchenne muscular dystrophy: implication for early diagnosis. J Pediatr 165 (2014), 1008–1010.
-
(2014)
J Pediatr
, vol.165
, pp. 1008-1010
-
-
Mirski, K.T.1
Crawford, T.O.2
-
20
-
-
84876669144
-
Improved detection of deletions and duplications in the DMD gene using the multiplex ligation-dependent probe amplification (MLPA) method
-
Sansović I, Barišić I, Dumić K, Improved detection of deletions and duplications in the DMD gene using the multiplex ligation-dependent probe amplification (MLPA) method. Biochem Genet 51 (2013), 189–201.
-
(2013)
Biochem Genet
, vol.51
, pp. 189-201
-
-
Sansović, I.1
Barišić, I.2
Dumić, K.3
-
21
-
-
51549094171
-
Microarray-based mutation detection in the dystrophin gene
-
Hegde, MR, Chin, EL, Mulle, JG, Okou, DT, Warren, ST, Zwick, ME, Microarray-based mutation detection in the dystrophin gene. Hum Mutat 29 (2008), 1091–1099.
-
(2008)
Hum Mutat
, vol.29
, pp. 1091-1099
-
-
Hegde, M.R.1
Chin, E.L.2
Mulle, J.G.3
Okou, D.T.4
Warren, S.T.5
Zwick, M.E.6
-
22
-
-
84922772691
-
A comprehensive genomic approach for neuromuscular diseases gives a high diagnostic yield
-
Ankala, A, da Silva, C, Gualandi, F, et al. A comprehensive genomic approach for neuromuscular diseases gives a high diagnostic yield. Ann Neurol 77 (2015), 206–214.
-
(2015)
Ann Neurol
, vol.77
, pp. 206-214
-
-
Ankala, A.1
da Silva, C.2
Gualandi, F.3
-
23
-
-
84910020266
-
Whole dystrophin gene analysis by next-generation sequencing: a comprehensive genetic diagnosis of Duchenne and Becker muscular dystrophy
-
Wang, Y, Yang, Y, Liu, J, et al. Whole dystrophin gene analysis by next-generation sequencing: a comprehensive genetic diagnosis of Duchenne and Becker muscular dystrophy. Mol Genet Genomics 289 (2014), 1013–1021.
-
(2014)
Mol Genet Genomics
, vol.289
, pp. 1013-1021
-
-
Wang, Y.1
Yang, Y.2
Liu, J.3
-
24
-
-
84890795613
-
Targeted next-generation sequencing as a comprehensive test for patients with and female carriers of DMD/BMD: a multi-population diagnostic study
-
Wei, X, Dai, Y, Yu, P, et al. Targeted next-generation sequencing as a comprehensive test for patients with and female carriers of DMD/BMD: a multi-population diagnostic study. Eur J Hum Genet 22 (2014), 110–118.
-
(2014)
Eur J Hum Genet
, vol.22
, pp. 110-118
-
-
Wei, X.1
Dai, Y.2
Yu, P.3
-
25
-
-
84976553960
-
Genetic diagnosis of Duchenne/Becker muscular dystrophy using next-generation sequencing: validation analysis of DMD mutations
-
Okubo, M, Minami, N, Goto, K, et al. Genetic diagnosis of Duchenne/Becker muscular dystrophy using next-generation sequencing: validation analysis of DMD mutations. J Hum Genet 61 (2016), 483–489.
-
(2016)
J Hum Genet
, vol.61
, pp. 483-489
-
-
Okubo, M.1
Minami, N.2
Goto, K.3
-
26
-
-
85042010554
-
Opinion 2.138—genetic testing of children
-
(accessed March 28, 2016).
-
American Medical Association. Opinion 2.138—genetic testing of children. http://www.ama-assn.org/ama/pub/physician-resources/medical-ethics/code-medical-ethics/opinion2138.page, 1996 (accessed March 28, 2016).
-
(1996)
-
-
-
27
-
-
0017129339
-
Screening for Duchenne muscular dystrophy
-
Dubowitz, V, Screening for Duchenne muscular dystrophy. Arch Dis Child 51 (1976), 249–251.
-
(1976)
Arch Dis Child
, vol.51
, pp. 249-251
-
-
Dubowitz, V.1
-
28
-
-
84880947740
-
195th ENMC International Workshop: Newborn screening for Duchenne muscular dystrophy 14-16th December, 2012, Naarden, The Netherlands
-
Ellis, JA, Vroom, E, Muntoni, F, 195th ENMC International Workshop: Newborn screening for Duchenne muscular dystrophy 14-16th December, 2012, Naarden, The Netherlands. Neuromuscul Disord 23 (2013), 682–689.
-
(2013)
Neuromuscul Disord
, vol.23
, pp. 682-689
-
-
Ellis, J.A.1
Vroom, E.2
Muntoni, F.3
-
29
-
-
33747591403
-
Newborn screening: toward a uniform screening panel and system
-
Watson, MS, Mann, MY, Lloyd-Puryear, MA, Rinaldo, P, Howell, RR, Newborn screening: toward a uniform screening panel and system. Genet Med 8:suppl (2006), 1S–11S.
-
(2006)
Genet Med
, vol.8
, pp. 1S-11S
-
-
Watson, M.S.1
Mann, M.Y.2
Lloyd-Puryear, M.A.3
Rinaldo, P.4
Howell, R.R.5
-
30
-
-
84900494729
-
Parental attitudes toward newborn screening for Duchenne/Becker muscular dystrophy and spinal muscular atrophy
-
Wood, MF, Hughes, SC, Hache, LP, et al. Parental attitudes toward newborn screening for Duchenne/Becker muscular dystrophy and spinal muscular atrophy. Muscle Nerve 49 (2014), 822–828.
-
(2014)
Muscle Nerve
, vol.49
, pp. 822-828
-
-
Wood, M.F.1
Hughes, S.C.2
Hache, L.P.3
-
31
-
-
84961119092
-
Twenty-year follow-up of newborn screening for patients with muscular dystrophy
-
Chung, J, Smith, AL, Hughes, SC, et al. Twenty-year follow-up of newborn screening for patients with muscular dystrophy. Muscle Nerve 53 (2016), 570–578.
-
(2016)
Muscle Nerve
, vol.53
, pp. 570-578
-
-
Chung, J.1
Smith, A.L.2
Hughes, S.C.3
-
32
-
-
84879902003
-
The 6-minute walk test and person-reported outcomes in boys with Duchenne muscular dystrophy and typically developing controls: longitudinal comparisons and clinically-meaningful changes over one year
-
Henricson, E, Abresch, R, Han, JJ, et al. The 6-minute walk test and person-reported outcomes in boys with Duchenne muscular dystrophy and typically developing controls: longitudinal comparisons and clinically-meaningful changes over one year. PLoS Curr, 2013, 5.
-
(2013)
PLoS Curr
, pp. 5
-
-
Henricson, E.1
Abresch, R.2
Han, J.J.3
-
33
-
-
84872223337
-
24 month longitudinal data in ambulant boys with Duchenne muscular dystrophy
-
Mazzone, ES, Pane, M, Sormani, MP, et al. 24 month longitudinal data in ambulant boys with Duchenne muscular dystrophy. PLoS One, 8, 2013, e52512.
-
(2013)
PLoS One
, vol.8
, pp. e52512
-
-
Mazzone, E.S.1
Pane, M.2
Sormani, M.P.3
-
34
-
-
84885183817
-
Detecting meaningful change using the North Star Ambulatory Assessment in Duchenne muscular dystrophy
-
Mayhew, AG, Cano, SJ, Scott, E, et al., the North Star Clinical Network for Neuromuscular Disease. Detecting meaningful change using the North Star Ambulatory Assessment in Duchenne muscular dystrophy. Dev Med Child Neurol 55 (2013), 1046–1052.
-
(2013)
Dev Med Child Neurol
, vol.55
, pp. 1046-1052
-
-
Mayhew, A.G.1
Cano, S.J.2
Scott, E.3
-
35
-
-
84883055833
-
The 6-minute walk test and other endpoints in Duchenne muscular dystrophy: longitudinal natural history observations over 48 weeks from a multicenter study
-
McDonald, CM, Henricson, EK, Abresch, RT, et al. The 6-minute walk test and other endpoints in Duchenne muscular dystrophy: longitudinal natural history observations over 48 weeks from a multicenter study. Muscle Nerve 48 (2013), 343–356.
-
(2013)
Muscle Nerve
, vol.48
, pp. 343-356
-
-
McDonald, C.M.1
Henricson, E.K.2
Abresch, R.T.3
-
36
-
-
85042025917
-
The PJ Nicholoff Steroid Protocol for Duchenne and Becker muscular dystrophy and adrenal suppression
-
ecurrents.md.d18deef7dac96ed135e0dc8739917b6e.
-
Kinnett, K, Noritz, G, The PJ Nicholoff Steroid Protocol for Duchenne and Becker muscular dystrophy and adrenal suppression. PLoS Curr, 9, 2017 ecurrents.md.d18deef7dac96ed135e0dc8739917b6e.
-
(2017)
PLoS Curr
, vol.9
-
-
Kinnett, K.1
Noritz, G.2
-
37
-
-
84882267702
-
Glucocorticoid treatment for the prevention of scoliosis in children with Duchenne muscular dystrophy: long-term follow-up
-
Lebel, DE, Corston, JA, McAdam, LC, Biggar, WD, Alman, BA, Glucocorticoid treatment for the prevention of scoliosis in children with Duchenne muscular dystrophy: long-term follow-up. J Bone Joint Surg Am 95 (2013), 1057–1061.
-
(2013)
J Bone Joint Surg Am
, vol.95
, pp. 1057-1061
-
-
Lebel, D.E.1
Corston, J.A.2
McAdam, L.C.3
Biggar, W.D.4
Alman, B.A.5
-
38
-
-
84860896654
-
Early corticosteroid treatment in 4 Duchenne muscular dystrophy patients: 14-year follow-up
-
Merlini, L, Gennari, M, Malaspina, E, et al. Early corticosteroid treatment in 4 Duchenne muscular dystrophy patients: 14-year follow-up. Muscle Nerve 45 (2012), 796–802.
-
(2012)
Muscle Nerve
, vol.45
, pp. 796-802
-
-
Merlini, L.1
Gennari, M.2
Malaspina, E.3
-
39
-
-
84962094373
-
Corticosteroid treatment and growth patterns in ambulatory males with Duchenne muscular dystrophy
-
13.e3
-
Lamb, MM, West, NA, Ouyang, L, et al. Corticosteroid treatment and growth patterns in ambulatory males with Duchenne muscular dystrophy. J Pediatr, 173, 2016, 207 13.e3.
-
(2016)
J Pediatr
, vol.173
, pp. 207
-
-
Lamb, M.M.1
West, N.A.2
Ouyang, L.3
-
40
-
-
84879551364
-
Corticosteroids in Duchenne muscular dystrophy: major variations in practice
-
Griggs, RC, Herr, BE, Reha, A, et al. Corticosteroids in Duchenne muscular dystrophy: major variations in practice. Muscle Nerve 48 (2013), 27–31.
-
(2013)
Muscle Nerve
, vol.48
, pp. 27-31
-
-
Griggs, R.C.1
Herr, B.E.2
Reha, A.3
-
41
-
-
84947910049
-
Prednisone/prednisolone and deflazacort regimens in the CINRG Duchenne Natural History Study
-
Bello, L, Gordish-Dressman, H, Morgenroth, LP, et al., the CINRG Investigators. Prednisone/prednisolone and deflazacort regimens in the CINRG Duchenne Natural History Study. Neurology 85 (2015), 1048–1055.
-
(2015)
Neurology
, vol.85
, pp. 1048-1055
-
-
Bello, L.1
Gordish-Dressman, H.2
Morgenroth, L.P.3
-
42
-
-
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 77 (2011), 444–452.
-
(2011)
Neurology
, vol.77
, pp. 444-452
-
-
Escolar, D.M.1
Hache, L.P.2
Clemens, P.R.3
-
43
-
-
84927669185
-
Ataluren treatment of patients with nonsense mutation dystrophinopathy
-
Bushby, K, Finkel, R, Wong, B, et al., the PTC124-GD-007-DMD STUDY GROUP. Ataluren treatment of patients with nonsense mutation dystrophinopathy. Muscle Nerve 50 (2014), 477–487.
-
(2014)
Muscle Nerve
, vol.50
, pp. 477-487
-
-
Bushby, K.1
Finkel, R.2
Wong, B.3
-
44
-
-
85024501735
-
Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial
-
McDonald, CM, Campbell, C, Torricelli, RE, et al., the Clinical Evaluator Training Group and the ACT DMD Study Group. Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet 390 (2017), 1489–1498.
-
(2017)
Lancet
, vol.390
, pp. 1489-1498
-
-
McDonald, C.M.1
Campbell, C.2
Torricelli, R.E.3
-
45
-
-
84958106352
-
Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy
-
Mendell, JR, Goemans, N, Lowes, LP, et al., the Eteplirsen Study Group and Telethon Foundation DMD Italian Network. Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy. Ann Neurol 79 (2016), 257–271.
-
(2016)
Ann Neurol
, vol.79
, pp. 257-271
-
-
Mendell, J.R.1
Goemans, N.2
Lowes, L.P.3
-
46
-
-
85018735509
-
Pharmacological advances for treatment in Duchenne muscular dystrophy
-
Guiraud, S, Davies, KE, Pharmacological advances for treatment in Duchenne muscular dystrophy. Curr Opin Pharmacol 34 (2017), 36–48.
-
(2017)
Curr Opin Pharmacol
, vol.34
, pp. 36-48
-
-
Guiraud, S.1
Davies, K.E.2
-
47
-
-
84861702194
-
The Canadian experience with long-term deflazacort treatment in Duchenne muscular dystrophy
-
McAdam, LC, Mayo, AL, Alman, BA, Biggar, WD, The Canadian experience with long-term deflazacort treatment in Duchenne muscular dystrophy. Acta Myol 31 (2012), 16–20.
-
(2012)
Acta Myol
, vol.31
, pp. 16-20
-
-
McAdam, L.C.1
Mayo, A.L.2
Alman, B.A.3
Biggar, W.D.4
-
48
-
-
84879551615
-
The Cooperative International Neuromuscular Research Group Duchenne Natural History Study: glucocorticoid treatment preserves clinically meaningful functional milestones and reduces rate of disease progression as measured by manual muscle testing and other commonly used clinical trial outcome measures
-
Henricson, EK, Abresch, RT, Cnaan, A, et al., the CINRG Investigators. The Cooperative International Neuromuscular Research Group Duchenne Natural History Study: glucocorticoid treatment preserves clinically meaningful functional milestones and reduces rate of disease progression as measured by manual muscle testing and other commonly used clinical trial outcome measures. Muscle Nerve 48 (2013), 55–67.
-
(2013)
Muscle Nerve
, vol.48
, pp. 55-67
-
-
Henricson, E.K.1
Abresch, R.T.2
Cnaan, A.3
-
49
-
-
84878983799
-
Motor and cognitive assessment of infants and young boys with Duchenne muscular dystrophy: results from the Muscular Dystrophy Association DMD Clinical Research Network
-
Connolly, AM, Florence, JM, Cradock, MM, et al., the MDA DMD Clinical Research Network. Motor and cognitive assessment of infants and young boys with Duchenne muscular dystrophy: results from the Muscular Dystrophy Association DMD Clinical Research Network. Neuromuscul Disord 23 (2013), 529–539.
-
(2013)
Neuromuscul Disord
, vol.23
, pp. 529-539
-
-
Connolly, A.M.1
Florence, J.M.2
Cradock, M.M.3
-
50
-
-
84919683240
-
Suitability of North Star Ambulatory Assessment in young boys with Duchenne muscular dystrophy
-
De Sanctis, R, Pane, M, Sivo, S, et al. Suitability of North Star Ambulatory Assessment in young boys with Duchenne muscular dystrophy. Neuromuscul Disord 25 (2015), 14–18.
-
(2015)
Neuromuscul Disord
, vol.25
, pp. 14-18
-
-
De Sanctis, R.1
Pane, M.2
Sivo, S.3
-
51
-
-
84885175848
-
Development of the performance of the upper limb module for Duchenne muscular dystrophy
-
Mayhew, A, Mazzone, ES, Eagle, M, et al. Development of the performance of the upper limb module for Duchenne muscular dystrophy. Dev Med Child Neurol 55 (2013), 1038–1045.
-
(2013)
Dev Med Child Neurol
, vol.55
, pp. 1038-1045
-
-
Mayhew, A.1
Mazzone, E.S.2
Eagle, M.3
-
52
-
-
84938980667
-
Reliability and validity of active-seated: an outcome in dystrophinopathy
-
Lowes, LP, Alfano, LN, Crawfis, R, et al. Reliability and validity of active-seated: an outcome in dystrophinopathy. Muscle Nerve 52 (2015), 356–362.
-
(2015)
Muscle Nerve
, vol.52
, pp. 356-362
-
-
Lowes, L.P.1
Alfano, L.N.2
Crawfis, R.3
-
53
-
-
84962006746
-
The performance of the upper limb scores correlate with pulmonary function test measures and Egen Klassifikation scores in Duchenne muscular dystrophy
-
Lee, HN, Sawnani, H, Horn, PS, Rybalsky, I, Relucio, L, Wong, BL, The performance of the upper limb scores correlate with pulmonary function test measures and Egen Klassifikation scores in Duchenne muscular dystrophy. Neuromuscul Disord 26 (2016), 264–271.
-
(2016)
Neuromuscul Disord
, vol.26
, pp. 264-271
-
-
Lee, H.N.1
Sawnani, H.2
Horn, P.S.3
Rybalsky, I.4
Relucio, L.5
Wong, B.L.6
-
54
-
-
78651349751
-
Continuous monitoring and quantification of multiple parameters of daily physical activity in ambulatory Duchenne muscular dystrophy patients
-
Jeannet, PY, Aminian, K, Bloetzer, C, Najafi, B, Paraschiv-Ionescu, A, Continuous monitoring and quantification of multiple parameters of daily physical activity in ambulatory Duchenne muscular dystrophy patients. Eur J Paediatr Neurol 15 (2011), 40–47.
-
(2011)
Eur J Paediatr Neurol
, vol.15
, pp. 40-47
-
-
Jeannet, P.Y.1
Aminian, K.2
Bloetzer, C.3
Najafi, B.4
Paraschiv-Ionescu, A.5
-
55
-
-
79956006620
-
Moving towards meaningful measurement: Rasch analysis of the North Star Ambulatory Assessment in Duchenne muscular dystrophy
-
Mayhew, A, Cano, S, Scott, E, Eagle, M, Bushby, K, Muntoni, F, Moving towards meaningful measurement: Rasch analysis of the North Star Ambulatory Assessment in Duchenne muscular dystrophy. Dev Med Child Neurol 53 (2011), 535–542.
-
(2011)
Dev Med Child Neurol
, vol.53
, pp. 535-542
-
-
Mayhew, A.1
Cano, S.2
Scott, E.3
Eagle, M.4
Bushby, K.5
Muntoni, F.6
-
56
-
-
84883050768
-
The 6-minute walk test and other clinical endpoints in Duchenne muscular dystrophy: reliability, concurrent validity, and minimal clinically important differences from a multicenter study
-
McDonald, CM, Henricson, EK, Abresch, RT, et al. The 6-minute walk test and other clinical endpoints in Duchenne muscular dystrophy: reliability, concurrent validity, and minimal clinically important differences from a multicenter study. Muscle Nerve 48 (2013), 357–368.
-
(2013)
Muscle Nerve
, vol.48
, pp. 357-368
-
-
McDonald, C.M.1
Henricson, E.K.2
Abresch, R.T.3
-
57
-
-
80054680303
-
Upper limb function in adults with Duchenne muscular dystrophy
-
Bartels, B, Pangalila, RF, Bergen, MP, Cobben, NA, Stam, HJ, Roebroeck, ME, Upper limb function in adults with Duchenne muscular dystrophy. J Rehabil Med 43 (2011), 770–775.
-
(2011)
J Rehabil Med
, vol.43
, pp. 770-775
-
-
Bartels, B.1
Pangalila, R.F.2
Bergen, M.P.3
Cobben, N.A.4
Stam, H.J.5
Roebroeck, M.E.6
-
58
-
-
84894248468
-
Reliability of the Performance of Upper Limb assessment in Duchenne muscular dystrophy
-
Pane, M, Mazzone, ES, Fanelli, L, et al. Reliability of the Performance of Upper Limb assessment in Duchenne muscular dystrophy. Neuromuscul Disord 24 (2014), 201–206.
-
(2014)
Neuromuscul Disord
, vol.24
, pp. 201-206
-
-
Pane, M.1
Mazzone, E.S.2
Fanelli, L.3
-
59
-
-
84937526368
-
Prevalence of fatigue, pain, and affective disorders in adults with Duchenne muscular dystrophy and their associations with quality of life
-
Pangalila, RF, van den Bos, GA, Bartels, B, Bergen, M, Stam, HJ, Roebroeck, ME, Prevalence of fatigue, pain, and affective disorders in adults with Duchenne muscular dystrophy and their associations with quality of life. Arch Phys Med Rehabil 96 (2015), 1242–1247.
-
(2015)
Arch Phys Med Rehabil
, vol.96
, pp. 1242-1247
-
-
Pangalila, R.F.1
van den Bos, G.A.2
Bartels, B.3
Bergen, M.4
Stam, H.J.5
Roebroeck, M.E.6
-
60
-
-
77956323075
-
Classification of the gait patterns of boys with Duchenne muscular dystrophy and their relationship to function
-
Sienko Thomas, S, Buckon, CE, Nicorici, A, Bagley, A, McDonald, CM, Sussman, MD, Classification of the gait patterns of boys with Duchenne muscular dystrophy and their relationship to function. J Child Neurol 25 (2010), 1103–1109.
-
(2010)
J Child Neurol
, vol.25
, pp. 1103-1109
-
-
Sienko Thomas, S.1
Buckon, C.E.2
Nicorici, A.3
Bagley, A.4
McDonald, C.M.5
Sussman, M.D.6
-
61
-
-
85010754845
-
Can quantitative muscle strength and functional motor ability differentiate the influence of age and corticosteroids in ambulatory boys with Duchenne muscular dystrophy?
-
published online July 8.
-
Buckon, C, Sienko, S, Bagley, A, et al. Can quantitative muscle strength and functional motor ability differentiate the influence of age and corticosteroids in ambulatory boys with Duchenne muscular dystrophy?. PLoS Curr, 2016 published online July 8. DOI:10.1371/currents.md.1ced64dff945f8958221fddcd4ee60b0.
-
(2016)
PLoS Curr
-
-
Buckon, C.1
Sienko, S.2
Bagley, A.3
-
62
-
-
85041999265
-
Duchenne Regulatory Science Consortium meeting on disease progression modeling for Duchenne muscular dystrophy
-
Larkindale, J, Abresch, R, Aviles, E, et al. Duchenne Regulatory Science Consortium meeting on disease progression modeling for Duchenne muscular dystrophy. PLoS Curr, 2017, 9.
-
(2017)
PLoS Curr
, pp. 9
-
-
Larkindale, J.1
Abresch, R.2
Aviles, E.3
-
63
-
-
84880950715
-
Ambulatory capacity and disease progression as measured by the 6-minute-walk-distance in Duchenne muscular dystrophy subjects on daily corticosteroids
-
Goemans, N, van den Hauwe, M, Wilson, R, van Impe, A, Klingels, K, Buyse, G, Ambulatory capacity and disease progression as measured by the 6-minute-walk-distance in Duchenne muscular dystrophy subjects on daily corticosteroids. Neuromuscul Disord 23 (2013), 618–623.
-
(2013)
Neuromuscul Disord
, vol.23
, pp. 618-623
-
-
Goemans, N.1
van den Hauwe, M.2
Wilson, R.3
van Impe, A.4
Klingels, K.5
Buyse, G.6
-
64
-
-
84907487779
-
Long term natural history data in ambulant boys with Duchenne muscular dystrophy: 36-month changes
-
Pane, M, Mazzone, ES, Sivo, S, et al. Long term natural history data in ambulant boys with Duchenne muscular dystrophy: 36-month changes. PLoS One, 9, 2014, e108205.
-
(2014)
PLoS One
, vol.9
, pp. e108205
-
-
Pane, M.1
Mazzone, E.S.2
Sivo, S.3
-
65
-
-
84897449030
-
6 minute walk test in Duchenne MD patients with different mutations: 12 month changes
-
Pane, M, Mazzone, ES, Sormani, MP, et al. 6 minute walk test in Duchenne MD patients with different mutations: 12 month changes. PLoS One, 9, 2014, e83400.
-
(2014)
PLoS One
, vol.9
, pp. e83400
-
-
Pane, M.1
Mazzone, E.S.2
Sormani, M.P.3
-
66
-
-
84925643643
-
Genetic modifiers of ambulation in the Cooperative International Neuromuscular Research Group Duchenne Natural History Study
-
Bello, L, Kesari, A, Gordish-Dressman, H, et al. Genetic modifiers of ambulation in the Cooperative International Neuromuscular Research Group Duchenne Natural History Study. Ann Neurol 77 (2015), 684–696.
-
(2015)
Ann Neurol
, vol.77
, pp. 684-696
-
-
Bello, L.1
Kesari, A.2
Gordish-Dressman, H.3
-
67
-
-
84991327511
-
Individualized prediction of changes in 6-minute walk distance for patients with Duchenne muscular dystrophy
-
Goemans, N, Vanden Hauwe, M, Signorovitch, J, Swallow, E, Song, J, the Collaborative Trajectory Analysis Project (cTAP). Individualized prediction of changes in 6-minute walk distance for patients with Duchenne muscular dystrophy. PLoS One, 11, 2016, e0164684.
-
(2016)
PLoS One
, vol.11
, pp. e0164684
-
-
Goemans, N.1
Vanden Hauwe, M.2
Signorovitch, J.3
Swallow, E.4
Song, J.5
-
68
-
-
84958863314
-
The NorthStar Ambulatory Assessment in Duchenne muscular dystrophy: considerations for the design of clinical trials
-
Ricotti, V, Ridout, DA, Pane, M, et al., the UK NorthStar Clinical Network. The NorthStar Ambulatory Assessment in Duchenne muscular dystrophy: considerations for the design of clinical trials. J Neurol Neurosurg Psychiatry 87 (2016), 149–155.
-
(2016)
J Neurol Neurosurg Psychiatry
, vol.87
, pp. 149-155
-
-
Ricotti, V.1
Ridout, D.A.2
Pane, M.3
-
69
-
-
84949486403
-
Health-related quality of life in patients with Duchenne muscular dystrophy: a multinational, cross-sectional study
-
Landfeldt, E, Lindgren, P, Bell, CF, et al. Health-related quality of life in patients with Duchenne muscular dystrophy: a multinational, cross-sectional study. Dev Med Child Neurol 58 (2016), 508–515.
-
(2016)
Dev Med Child Neurol
, vol.58
, pp. 508-515
-
-
Landfeldt, E.1
Lindgren, P.2
Bell, C.F.3
-
70
-
-
0028445695
-
Preliminary validation of a new instrument to measure life satisfaction in adolescents with neuromuscular disorders
-
Reid, DT, Renwick, RM, Preliminary validation of a new instrument to measure life satisfaction in adolescents with neuromuscular disorders. Int J Rehabil Res 17 (1994), 184–188.
-
(1994)
Int J Rehabil Res
, vol.17
, pp. 184-188
-
-
Reid, D.T.1
Renwick, R.M.2
-
71
-
-
0034993579
-
Relating familial stress to the psychosocial adjustment of adolescents with Duchenne muscular dystrophy
-
Reid, DT, Renwick, RM, Relating familial stress to the psychosocial adjustment of adolescents with Duchenne muscular dystrophy. Int J Rehabil Res 24 (2001), 83–93.
-
(2001)
Int J Rehabil Res
, vol.24
, pp. 83-93
-
-
Reid, D.T.1
Renwick, R.M.2
-
72
-
-
84959041053
-
Quality improvement in neurology: muscular dystrophy quality measures
-
Narayanaswami, P, Dubinsky, R, Wang, D, et al. Quality improvement in neurology: muscular dystrophy quality measures. Neurology 85 (2015), 905–909.
-
(2015)
Neurology
, vol.85
, pp. 905-909
-
-
Narayanaswami, P.1
Dubinsky, R.2
Wang, D.3
-
73
-
-
84901030899
-
One year outcome of boys with Duchenne muscular dystrophy using the Bayley-III scales of infant and toddler development
-
Connolly, AM, Florence, JM, Cradock, MM, et al. One year outcome of boys with Duchenne muscular dystrophy using the Bayley-III scales of infant and toddler development. Pediatr Neurol 50 (2014), 557–563.
-
(2014)
Pediatr Neurol
, vol.50
, pp. 557-563
-
-
Connolly, A.M.1
Florence, J.M.2
Cradock, M.M.3
-
74
-
-
84983465461
-
Revised North Star Ambulatory Assessment for young boys with Duchenne muscular dystrophy
-
Mercuri, E, Coratti, G, Messina, S, et al. Revised North Star Ambulatory Assessment for young boys with Duchenne muscular dystrophy. PLoS One, 11, 2016, e0160195.
-
(2016)
PLoS One
, vol.11
, pp. e0160195
-
-
Mercuri, E.1
Coratti, G.2
Messina, S.3
-
75
-
-
84971372948
-
Hip kinetics during gait are clinically meaningful outcomes in young boys with Duchenne muscular dystrophy
-
Heberer, K, Fowler, E, Staudt, L, et al. Hip kinetics during gait are clinically meaningful outcomes in young boys with Duchenne muscular dystrophy. Gait Posture 48 (2016), 159–164.
-
(2016)
Gait Posture
, vol.48
, pp. 159-164
-
-
Heberer, K.1
Fowler, E.2
Staudt, L.3
-
76
-
-
0004105372
-
Motor assessment of the developing infant
-
Saunders
-
Piper, MC, Darrah, J, Motor assessment of the developing infant. 1994, Saunders.
-
(1994)
-
-
Piper, M.C.1
Darrah, J.2
-
77
-
-
84866732900
-
Attention deficit hyperactivity disorder and cognitive function in Duchenne muscular dystrophy: phenotype-genotype correlation
-
09.e1
-
Pane, M, Lombardo, ME, Alfieri, P, et al. Attention deficit hyperactivity disorder and cognitive function in Duchenne muscular dystrophy: phenotype-genotype correlation. J Pediatr, 161, 2012, 705 09.e1.
-
(2012)
J Pediatr
, vol.161
, pp. 705
-
-
Pane, M.1
Lombardo, M.E.2
Alfieri, P.3
-
78
-
-
36348945650
-
Behavior patterns in Duchenne muscular dystrophy: report on the Parent Project Muscular Dystrophy behavior workshop 8–9 of December 2006, Philadelphia, USA
-
Poysky, J, the Behavior in DMD Study Group. Behavior patterns in Duchenne muscular dystrophy: report on the Parent Project Muscular Dystrophy behavior workshop 8–9 of December 2006, Philadelphia, USA. Neuromuscul Disord 17 (2007), 986–994.
-
(2007)
Neuromuscul Disord
, vol.17
, pp. 986-994
-
-
Poysky, J.1
-
79
-
-
84986538695
-
Clinical trial readiness in non-ambulatory boys and men with Duchenne muscular dystrophy: MDA-DMD network follow-up
-
Connolly, AM, Florence, JM, Zaidman, CM, et al., the MDA-DMD Clinical Research Network. Clinical trial readiness in non-ambulatory boys and men with Duchenne muscular dystrophy: MDA-DMD network follow-up. Muscle Nerve 54 (2016), 681–689.
-
(2016)
Muscle Nerve
, vol.54
, pp. 681-689
-
-
Connolly, A.M.1
Florence, J.M.2
Zaidman, C.M.3
-
80
-
-
84925350947
-
Outcome reliability in non-ambulatory boys/men with Duchenne muscular dystrophy
-
Connolly, AM, Malkus, EC, Mendell, JR, et al., the MDA DMD Clinical Research Network. Outcome reliability in non-ambulatory boys/men with Duchenne muscular dystrophy. Muscle Nerve 51 (2015), 522–532.
-
(2015)
Muscle Nerve
, vol.51
, pp. 522-532
-
-
Connolly, A.M.1
Malkus, E.C.2
Mendell, J.R.3
-
81
-
-
84961118524
-
Reachable workspace and performance of upper limb (PUL) in Duchenne muscular dystrophy
-
Han, JJ, de Bie, E, Nicorici, A, et al. Reachable workspace and performance of upper limb (PUL) in Duchenne muscular dystrophy. Muscle Nerve 53 (2016), 545–554.
-
(2016)
Muscle Nerve
, vol.53
, pp. 545-554
-
-
Han, J.J.1
de Bie, E.2
Nicorici, A.3
-
82
-
-
80051707432
-
Serial casting for the management of ankle contracture in Duchenne muscular dystrophy
-
Glanzman, AM, Flickinger, JM, Dholakia, KH, Bönnemann, CG, Finkel, RS, Serial casting for the management of ankle contracture in Duchenne muscular dystrophy. Pediatr Phys Ther 23 (2011), 275–279.
-
(2011)
Pediatr Phys Ther
, vol.23
, pp. 275-279
-
-
Glanzman, A.M.1
Flickinger, J.M.2
Dholakia, K.H.3
Bönnemann, C.G.4
Finkel, R.S.5
-
83
-
-
84988637152
-
Muscular dystrophy and spinal muscular atrophy
-
SK Campbell RJ Palisano MN Orlin Elsevier Saunders St Louis, MO
-
Stuberg, WA, Muscular dystrophy and spinal muscular atrophy. Campbell, SK, Palisano, RJ, Orlin, MN, (eds.) Physical therapy for children, 2012, Elsevier Saunders, St Louis, MO, 353–384.
-
(2012)
Physical therapy for children
, pp. 353-384
-
-
Stuberg, W.A.1
-
84
-
-
82455162342
-
Combination of steroids and ischial weight-bearing knee ankle foot orthoses in Duchenne's muscular dystrophy prolongs ambulation past 20 years of age—a case report
-
Pardo, AC, Do, T, Ryder, T, Meyer, A, Miles, L, Wong, BL, Combination of steroids and ischial weight-bearing knee ankle foot orthoses in Duchenne's muscular dystrophy prolongs ambulation past 20 years of age—a case report. Neuromuscul Disord 21 (2011), 800–802.
-
(2011)
Neuromuscul Disord
, vol.21
, pp. 800-802
-
-
Pardo, A.C.1
Do, T.2
Ryder, T.3
Meyer, A.4
Miles, L.5
Wong, B.L.6
-
85
-
-
33749122721
-
Knee-ankle-foot orthosis in children with Duchenne muscular dystrophy: user views and adjustment
-
Garralda, ME, Muntoni, F, Cunniff, A, Caneja, AD, Knee-ankle-foot orthosis in children with Duchenne muscular dystrophy: user views and adjustment. Eur J Paediatr Neurol 10 (2006), 186–191.
-
(2006)
Eur J Paediatr Neurol
, vol.10
, pp. 186-191
-
-
Garralda, M.E.1
Muntoni, F.2
Cunniff, A.3
Caneja, A.D.4
-
86
-
-
84857099915
-
Cardiac involvement in muscular dystrophy: advances in diagnosis and therapy
-
Yilmaz, A, Sechtem, U, Cardiac involvement in muscular dystrophy: advances in diagnosis and therapy. Heart 98 (2012), 420–429.
-
(2012)
Heart
, vol.98
, pp. 420-429
-
-
Yilmaz, A.1
Sechtem, U.2
-
87
-
-
84938095654
-
Cardiopulmonary exercise testing in children and adolescents with dystrophinopathies: a pilot study
-
Bartels, B, Takken, T, Blank, AC, van Moorsel, H, van der Pol, WL, de Groot, JF, Cardiopulmonary exercise testing in children and adolescents with dystrophinopathies: a pilot study. Pediatr Phys Ther 27 (2015), 227–234.
-
(2015)
Pediatr Phys Ther
, vol.27
, pp. 227-234
-
-
Bartels, B.1
Takken, T.2
Blank, A.C.3
van Moorsel, H.4
van der Pol, W.L.5
de Groot, J.F.6
-
88
-
-
84938077743
-
Commentary on “Cardiopulmonary exercise testing in children and adolescents with dystrophinopathies: a pilot study”
-
Case, LE, Hartzell, AS, Commentary on “Cardiopulmonary exercise testing in children and adolescents with dystrophinopathies: a pilot study”. Pediatr Phys Ther, 27, 2015, 235.
-
(2015)
Pediatr Phys Ther
, vol.27
, pp. 235
-
-
Case, L.E.1
Hartzell, A.S.2
-
89
-
-
84928411365
-
Nitric oxide synthase deficiency and the pathophysiology of muscular dystrophy
-
Tidball, JG, Wehling-Henricks, M, Nitric oxide synthase deficiency and the pathophysiology of muscular dystrophy. J Physiol 592 (2014), 4627–4638.
-
(2014)
J Physiol
, vol.592
, pp. 4627-4638
-
-
Tidball, J.G.1
Wehling-Henricks, M.2
-
90
-
-
84865567343
-
Exercise in neuromuscular diseases
-
Abresch, RT, Carter, GT, Han, JJ, McDonald, CM, Exercise in neuromuscular diseases. Phys Med Rehabil Clin N Am 23 (2012), 653–673.
-
(2012)
Phys Med Rehabil Clin N Am
, vol.23
, pp. 653-673
-
-
Abresch, R.T.1
Carter, G.T.2
Han, J.J.3
McDonald, C.M.4
-
91
-
-
84928214116
-
Different types of upper extremity exercise training in Duchenne muscular dystrophy: effects on functional performance, strength, endurance, and ambulation
-
Alemdaroğlu, I, Karaduman, A, Yilmaz, OT, Topaloğlu, H, Different types of upper extremity exercise training in Duchenne muscular dystrophy: effects on functional performance, strength, endurance, and ambulation. Muscle Nerve 51 (2015), 697–705.
-
(2015)
Muscle Nerve
, vol.51
, pp. 697-705
-
-
Alemdaroğlu, I.1
Karaduman, A.2
Yilmaz, O.T.3
Topaloğlu, H.4
-
92
-
-
84885404212
-
Assisted bicycle training delays functional deterioration in boys with Duchenne muscular dystrophy: the randomized controlled trial “no use is disuse”
-
Jansen, M, van Alfen, N, Geurts, AC, de Groot, IJ, Assisted bicycle training delays functional deterioration in boys with Duchenne muscular dystrophy: the randomized controlled trial “no use is disuse”. Neurorehabil Neural Repair 27 (2013), 816–827.
-
(2013)
Neurorehabil Neural Repair
, vol.27
, pp. 816-827
-
-
Jansen, M.1
van Alfen, N.2
Geurts, A.C.3
de Groot, I.J.4
-
93
-
-
84948665463
-
Back pain in Duchenne muscular dystrophy: steroids are not always the culprit
-
Segal, LS, Odgers, R, Carpentieri, D, Shrader, MW, Back pain in Duchenne muscular dystrophy: steroids are not always the culprit. J Pediatr Orthop B 25 (2016), 81–85.
-
(2016)
J Pediatr Orthop B
, vol.25
, pp. 81-85
-
-
Segal, L.S.1
Odgers, R.2
Carpentieri, D.3
Shrader, M.W.4
-
94
-
-
79952740316
-
Endocrine aspects of Duchenne muscular dystrophy
-
Bianchi, ML, Biggar, D, Bushby, K, Rogol, AD, Rutter, MM, Tseng, B, Endocrine aspects of Duchenne muscular dystrophy. Neuromuscul Disord 21 (2011), 298–303.
-
(2011)
Neuromuscul Disord
, vol.21
, pp. 298-303
-
-
Bianchi, M.L.1
Biggar, D.2
Bushby, K.3
Rogol, A.D.4
Rutter, M.M.5
Tseng, B.6
-
95
-
-
79960911228
-
Report on the Second Endocrine Aspects Of Duchenne Muscular Dystrophy Conference December 1–2, 2010, Baltimore, Maryland, USA
-
Leung, DG, Germain-Lee, EL, Denger, BE, Wagner, KR, Report on the Second Endocrine Aspects Of Duchenne Muscular Dystrophy Conference December 1–2, 2010, Baltimore, Maryland, USA. Neuromuscul Disord 21 (2011), 594–601.
-
(2011)
Neuromuscul Disord
, vol.21
, pp. 594-601
-
-
Leung, D.G.1
Germain-Lee, E.L.2
Denger, B.E.3
Wagner, K.R.4
-
96
-
-
84952638700
-
Short stature and pubertal delay in Duchenne muscular dystrophy
-
Wood, CL, Straub, V, Guglieri, M, Bushby, K, Cheetham, T, Short stature and pubertal delay in Duchenne muscular dystrophy. Arch Dis Child 101 (2016), 101–106.
-
(2016)
Arch Dis Child
, vol.101
, pp. 101-106
-
-
Wood, C.L.1
Straub, V.2
Guglieri, M.3
Bushby, K.4
Cheetham, T.5
-
97
-
-
84888382319
-
Patterns of growth in ambulatory males with Duchenne muscular dystrophy
-
63.e1
-
West, NA, Yang, ML, Weitzenkamp, DA, et al. Patterns of growth in ambulatory males with Duchenne muscular dystrophy. J Pediatr, 163, 2013, 1759 63.e1.
-
(2013)
J Pediatr
, vol.163
, pp. 1759
-
-
West, N.A.1
Yang, M.L.2
Weitzenkamp, D.A.3
-
98
-
-
0004165071
-
Anthropometric standardization reference manual
-
Human Kinetic Books Champaign, IL
-
Lohman, TG, Roche, AF, Martorell, R, Anthropometric standardization reference manual. 1988, Human Kinetic Books, Champaign, IL.
-
(1988)
-
-
Lohman, T.G.1
Roche, A.F.2
Martorell, R.3
-
99
-
-
84870393322
-
Growth hormone treatment in boys with Duchenne muscular dystrophy and glucocorticoid-induced growth failure
-
Rutter, MM, Collins, J, Rose, SR, et al. Growth hormone treatment in boys with Duchenne muscular dystrophy and glucocorticoid-induced growth failure. Neuromuscul Disord 22 (2012), 1046–1056.
-
(2012)
Neuromuscul Disord
, vol.22
, pp. 1046-1056
-
-
Rutter, M.M.1
Collins, J.2
Rose, S.R.3
-
100
-
-
0023831330
-
Relation between height and clinical course in Duchenne muscular dystrophy
-
Zatz, M, Rapaport, D, Vainzof, M, et al. Relation between height and clinical course in Duchenne muscular dystrophy. Am J Med Genet 29 (1988), 405–410.
-
(1988)
Am J Med Genet
, vol.29
, pp. 405-410
-
-
Zatz, M.1
Rapaport, D.2
Vainzof, M.3
-
101
-
-
84883052548
-
Why short stature is beneficial in Duchenne muscular dystrophy
-
Bodor, M, McDonald, CM, Why short stature is beneficial in Duchenne muscular dystrophy. Muscle Nerve 48 (2013), 336–342.
-
(2013)
Muscle Nerve
, vol.48
, pp. 336-342
-
-
Bodor, M.1
McDonald, C.M.2
-
102
-
-
77954409942
-
Testosterone therapy in men with androgen deficiency syndromes: an Endocrine Society clinical practice guideline
-
Bhasin, S, Cunningham, GR, Hayes, FJ, et al. Testosterone therapy in men with androgen deficiency syndromes: an Endocrine Society clinical practice guideline. J Clin Endocrinol Metab 95 (2010), 2536–2559.
-
(2010)
J Clin Endocrinol Metab
, vol.95
, pp. 2536-2559
-
-
Bhasin, S.1
Cunningham, G.R.2
Hayes, F.J.3
-
103
-
-
84948075506
-
Testosterone treatment of pubertal delay in Duchenne muscular dystrophy
-
Wood, CL, Cheetham, TD, Guglieri, M, et al. Testosterone treatment of pubertal delay in Duchenne muscular dystrophy. Neuropediatrics 46 (2015), 371–376.
-
(2015)
Neuropediatrics
, vol.46
, pp. 371-376
-
-
Wood, C.L.1
Cheetham, T.D.2
Guglieri, M.3
-
104
-
-
84930787882
-
Adrenal insufficiency in corticosteroids use: systematic review and meta-analysis
-
Broersen, LH, Pereira, AM, Jørgensen, JO, Dekkers, OM, Adrenal insufficiency in corticosteroids use: systematic review and meta-analysis. J Clin Endocrinol Metab 100 (2015), 2171–2180.
-
(2015)
J Clin Endocrinol Metab
, vol.100
, pp. 2171-2180
-
-
Broersen, L.H.1
Pereira, A.M.2
Jørgensen, J.O.3
Dekkers, O.M.4
-
105
-
-
78049507370
-
Clinical Review: the diagnosis and management of central hypoadrenalism
-
Grossman, AB, Clinical Review: the diagnosis and management of central hypoadrenalism. J Clin Endocrinol Metab 95 (2010), 4855–4863.
-
(2010)
J Clin Endocrinol Metab
, vol.95
, pp. 4855-4863
-
-
Grossman, A.B.1
-
106
-
-
84928214661
-
Reduced mandibular range of motion in Duchenne muscular dystrophy: predictive factors
-
van Bruggen, HW, Van Den Engel-Hoek, L, Steenks, MH, et al. Reduced mandibular range of motion in Duchenne muscular dystrophy: predictive factors. J Oral Rehabil 42 (2015), 430–438.
-
(2015)
J Oral Rehabil
, vol.42
, pp. 430-438
-
-
van Bruggen, H.W.1
Van Den Engel-Hoek, L.2
Steenks, M.H.3
-
107
-
-
84856953421
-
Decreased resting energy expenditure in patients with Duchenne muscular dystrophy
-
Shimizu-Fujiwara, M, Komaki, H, Nakagawa, E, et al. Decreased resting energy expenditure in patients with Duchenne muscular dystrophy. Brain Dev 34 (2012), 206–212.
-
(2012)
Brain Dev
, vol.34
, pp. 206-212
-
-
Shimizu-Fujiwara, M.1
Komaki, H.2
Nakagawa, E.3
-
108
-
-
85032179658
-
Dietary guidelines for Americans 2015–2020
-
8th edn. (accessed Dec 20, 2017).
-
US Department of Health and Human Services and US Department of Agriculture. Dietary guidelines for Americans 2015–2020. 8th edn. https://health.gov/dietaryguidelines/2015/guidelines/#navigation, 2015 (accessed Dec 20, 2017).
-
(2015)
-
-
-
109
-
-
84941732305
-
Treatable renal failure found in non-ambulatory Duchenne muscular dystrophy patients
-
Motoki, T, Shimizu-Motohashi, Y, Komaki, H, et al. Treatable renal failure found in non-ambulatory Duchenne muscular dystrophy patients. Neuromuscul Disord 25 (2015), 754–757.
-
(2015)
Neuromuscul Disord
, vol.25
, pp. 754-757
-
-
Motoki, T.1
Shimizu-Motohashi, Y.2
Komaki, H.3
-
110
-
-
85042004966
-
Dietary reference intakes—the essential guide to nutrient requirements
-
The National Academies Press Washington, DC
-
Institute of Medicine. Dietary reference intakes—the essential guide to nutrient requirements. 2006, The National Academies Press, Washington, DC.
-
(2006)
-
-
-
111
-
-
84867097184
-
Predicting resting energy expenditure in boys with Duchenne muscular dystrophy
-
Elliott, SA, Davidson, ZE, Davies, PS, Truby, H, Predicting resting energy expenditure in boys with Duchenne muscular dystrophy. Eur J Paediatr Neurol 16 (2012), 631–635.
-
(2012)
Eur J Paediatr Neurol
, vol.16
, pp. 631-635
-
-
Elliott, S.A.1
Davidson, Z.E.2
Davies, P.S.3
Truby, H.4
-
112
-
-
0000441509
-
The maintenance need for water in parenteral fluid therapy
-
Holliday, MA, Segar, WE, The maintenance need for water in parenteral fluid therapy. Pediatrics 19 (1957), 823–832.
-
(1957)
Pediatrics
, vol.19
, pp. 823-832
-
-
Holliday, M.A.1
Segar, W.E.2
-
113
-
-
33947588606
-
-
National Academies Press Washington DC
-
Institute of Medicine of the National Academies. Otten, JJ, Hellwig, JP, Meyers, LD, (eds.) Dietary reference intakes: the essential guide to nutrient requirements, 2006, National Academies Press, Washington DC.
-
(2006)
Dietary reference intakes: the essential guide to nutrient requirements
-
-
Otten, J.J.1
Hellwig, J.P.2
Meyers, L.D.3
-
114
-
-
78650918917
-
-
The National Academies Press Washington, DC
-
Institute of Medicine. Ross, AC, Taylor, CL, Yaktine, AL, Valle, HBD, (eds.) Dietary reference intakes for calcium and vitamin D, 2011, The National Academies Press, Washington, DC.
-
(2011)
Dietary reference intakes for calcium and vitamin D
-
-
Ross, A.C.1
Taylor, C.L.2
Yaktine, A.L.3
Valle, H.B.D.4
-
115
-
-
79956024161
-
Natural evolution of weight status in Duchenne muscular dystrophy: a retrospective audit
-
Martigne, L, Salleron, J, Mayer, M, et al. Natural evolution of weight status in Duchenne muscular dystrophy: a retrospective audit. Br J Nutr 105 (2011), 1486–1491.
-
(2011)
Br J Nutr
, vol.105
, pp. 1486-1491
-
-
Martigne, L.1
Salleron, J.2
Mayer, M.3
-
116
-
-
84937901049
-
Nutrition Considerations in Duchenne muscular dystrophy
-
Davis, J, Samuels, E, Mullins, L, Nutrition Considerations in Duchenne muscular dystrophy. Nutr Clin Pract 30 (2015), 511–521.
-
(2015)
Nutr Clin Pract
, vol.30
, pp. 511-521
-
-
Davis, J.1
Samuels, E.2
Mullins, L.3
-
117
-
-
85042028603
-
[Pediatric Obesity]
-
7th edn. American Academy of Pediatrics Elk Grove Village, IL
-
American Academy of Pediatrics Committee on Nutrition. [Pediatric Obesity]. Kleinman, RE, Greer, FR, (eds.) Pediatric nutrition, 7th edn., 2014, American Academy of Pediatrics, Elk Grove Village, IL, 827.
-
(2014)
Pediatric nutrition
, pp. 827
-
-
Kleinman, R.E.1
Greer, F.R.2
-
118
-
-
84953294474
-
Dysphagia in Duchenne muscular dystrophy: practical recommendations to guide management
-
Toussaint, M, Davidson, Z, Bouvoie, V, Evenepoel, N, Haan, J, Soudon, P, Dysphagia in Duchenne muscular dystrophy: practical recommendations to guide management. Disabil Rehabil 38 (2016), 2052–2062.
-
(2016)
Disabil Rehabil
, vol.38
, pp. 2052-2062
-
-
Toussaint, M.1
Davidson, Z.2
Bouvoie, V.3
Evenepoel, N.4
Haan, J.5
Soudon, P.6
-
119
-
-
84874823117
-
Dysphagia in Duchenne muscular dystrophy assessed by validated questionnaire
-
Archer, SK, Garrod, R, Hart, N, Miller, S, Dysphagia in Duchenne muscular dystrophy assessed by validated questionnaire. Int J Lang Commun Disord 48 (2013), 240–246.
-
(2013)
Int J Lang Commun Disord
, vol.48
, pp. 240-246
-
-
Archer, S.K.1
Garrod, R.2
Hart, N.3
Miller, S.4
-
120
-
-
41449085192
-
Dysphagia in patients with Duchenne muscular dystrophy evaluated with a questionnaire and videofluorography
-
Hanayama, K, Liu, M, Higuchi, Y, et al. Dysphagia in patients with Duchenne muscular dystrophy evaluated with a questionnaire and videofluorography. Disabil Rehabil 30 (2008), 517–522.
-
(2008)
Disabil Rehabil
, vol.30
, pp. 517-522
-
-
Hanayama, K.1
Liu, M.2
Higuchi, Y.3
-
121
-
-
75949124376
-
Efficacy and tolerance of gastrostomy feeding in Duchenne muscular dystrophy
-
Martigne, L, Seguy, D, Pellegrini, N, et al. Efficacy and tolerance of gastrostomy feeding in Duchenne muscular dystrophy. Clin Nutr 29 (2010), 60–64.
-
(2010)
Clin Nutr
, vol.29
, pp. 60-64
-
-
Martigne, L.1
Seguy, D.2
Pellegrini, N.3
-
122
-
-
84955591337
-
Constipation in Duchenne muscular dystrophy: prevalence, diagnosis, and treatment
-
Kraus, D, Wong, BL, Horn, PS, Kaul, A, Constipation in Duchenne muscular dystrophy: prevalence, diagnosis, and treatment. J Pediatr 171 (2016), 183–188.
-
(2016)
J Pediatr
, vol.171
, pp. 183-188
-
-
Kraus, D.1
Wong, B.L.2
Horn, P.S.3
Kaul, A.4
-
123
-
-
16844372638
-
Evolution of gastric electrical features and gastric emptying in children with Duchenne and Becker muscular dystrophy
-
Borrelli, O, Salvia, G, Mancini, V, et al. Evolution of gastric electrical features and gastric emptying in children with Duchenne and Becker muscular dystrophy. Am J Gastroenterol 100 (2005), 695–702.
-
(2005)
Am J Gastroenterol
, vol.100
, pp. 695-702
-
-
Borrelli, O.1
Salvia, G.2
Mancini, V.3
-
124
-
-
84957998974
-
Proton pump inhibitor use and the risk of chronic kidney disease
-
Lazarus, B, Chen, Y, Wilson, FP, et al. Proton pump inhibitor use and the risk of chronic kidney disease. JAMA Intern Med 176 (2016), 238–246.
-
(2016)
JAMA Intern Med
, vol.176
, pp. 238-246
-
-
Lazarus, B.1
Chen, Y.2
Wilson, F.P.3
-
125
-
-
84934943578
-
Risk of community-acquired pneumonia with outpatient proton-pump inhibitor therapy: a systematic review and meta-analysis
-
Lambert, AA, Lam, JO, Paik, JJ, Ugarte-Gil, C, Drummond, MB, Crowell, TA, Risk of community-acquired pneumonia with outpatient proton-pump inhibitor therapy: a systematic review and meta-analysis. PLoS One, 10, 2015, e0128004.
-
(2015)
PLoS One
, vol.10
, pp. e0128004
-
-
Lambert, A.A.1
Lam, J.O.2
Paik, J.J.3
Ugarte-Gil, C.4
Drummond, M.B.5
Crowell, T.A.6
-
126
-
-
84959432144
-
Non-invasive prenatal diagnosis of Duchenne and Becker muscular dystrophies by relative haplotype dosage
-
Parks, M, Court, S, Cleary, S, et al. Non-invasive prenatal diagnosis of Duchenne and Becker muscular dystrophies by relative haplotype dosage. Prenat Diagn 36 (2016), 312–320.
-
(2016)
Prenat Diagn
, vol.36
, pp. 312-320
-
-
Parks, M.1
Court, S.2
Cleary, S.3
-
127
-
-
85026383513
-
Longitudinal community walking activity in Duchenne muscular dystrophy
-
published online July 10.
-
Fowler, EG, Staudt, LA, Heberer, KR, et al. Longitudinal community walking activity in Duchenne muscular dystrophy. Muscle Nerve, 2017 published online July 10. DOI:10.1002/mus.25743.
-
(2017)
Muscle Nerve
-
-
Fowler, E.G.1
Staudt, L.A.2
Heberer, K.R.3
-
128
-
-
84964684031
-
Beneficial effects of ankle-foot orthosis daytime use on the gait of Duchenne muscular dystrophy patients
-
de Souza, MA, Figueiredo, MM, de Baptista, CR, Aldaves, RD, Mattiello-Sverzut, AC, Beneficial effects of ankle-foot orthosis daytime use on the gait of Duchenne muscular dystrophy patients. Clin Biomech (Bristol, Avon) 35 (2016), 102–110.
-
(2016)
Clin Biomech (Bristol, Avon)
, vol.35
, pp. 102-110
-
-
de Souza, M.A.1
Figueiredo, M.M.2
de Baptista, C.R.3
Aldaves, R.D.4
Mattiello-Sverzut, A.C.5
-
129
-
-
84959370487
-
Drugs in development and dietary approach for Duchenne muscular dystrophy
-
Angelini, C, Tasca, E, Drugs in development and dietary approach for Duchenne muscular dystrophy. Orphan Drugs Res Rev 5 (2015), 51–60.
-
(2015)
Orphan Drugs Res Rev
, vol.5
, pp. 51-60
-
-
Angelini, C.1
Tasca, E.2
|