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Multi-Omics Identifies Circulating miRNA and Protein Biomarkers for Facioscapulohumeral Dystrophy
Heier CR, Zhang A, Nguyen NY, et al.
Journal of personalized medicine, 2020, 10, 4, p 236
The CINRG Becker Natural History Study: Baseline Characteristics
Clemens PR, Niizawa G, Feng J, et al.
Muscle & Nerve, 2020
Randomized phase 2 trial and open-label extension of domagrozumab in Duchenne muscular dystrophy
Wagner KR, Abdel-Hamid HZ, Mah JK, et al.
Neuromuscular disorders : NMD, 2020, 30, 6, p 492
A national spinal muscular atrophy registry for real world evidence
Hodgkinson V, Oskoui M, Lounsberry J, et al.
The Canadian journal of neurological sciences. Le journal canadien des sciences neurologiques, 2020
Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A Phase 2 Randomized Clinical Trial
Clemens PR, Rao VK, Connolly AM, et al.
JAMA Neurology, 2020
Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy
Campbell C, Barohn RJ, Bertini E, et al.
Journal of comparative effectiveness research, 2020
The Canadian Neuromuscular Disease Registry 2010-2019: A Decade of Facilitating Clinical Research Through a Nationwide, Pan-Neuromuscular Disease Registry
Hodgkinson V, Lounsberry J, M'Dahoma S, et al.
Journal of Neuromuscular Diseases, 2020
Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study
Smith EC, Conklin LS, Hoffman EP, et al.
PLoS medicine, 2020, 17, 9
Disease-specific and glucocorticoid-responsive serum biomarkers for Duchenne Muscular Dystrophy
Hathout Y, Liang C, Ogundele M, et al.
Scientific Reports, 2019, 9, 1
Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function
Hoffman EP, Schwartz BD, Mengle-Gaw LJ, et al.
Neurology, 2019, 93, 13
A Pediatric Review of Facioscapulohumeral Muscular Dystrophy
Mah JK, Chen YW
Journal of pediatric neurology : JPN, 2018, 16, 4, p 222
Current and Emerging Therapies for Duchenne Muscular Dystrophy
Crone M, Crone M, Mah JK
Current treatment options in neurology, 2018, 20, 8
An Overview of Recent Therapeutics Advances for Duchenne Muscular Dystrophy
Mah JK
Methods in molecular biology, 2018, 1687, p 3
A multinational study on motor function in early-onset FSHD
Mah JK, Feng J, Jacobs MB, et al.
Neurology, 2018, 90, 15
The Canadian Neuromuscular Disease Registry: Connecting patients to national and international research opportunities
Wei Y, McCormick A, MacKenzie A, et al.
Paediatrics & child health, 2018, 23, 1, p 20
Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug
Conklin LS, Conklin LS, Damsker JM, et al.
Pharmacological Research, 2018, 136, p 140
A checklist for clinical trials in rare disease: obstacles and anticipatory actions-lessons learned from the FOR-DMD trial
Crow RA, Hart KA, McDermott MP, et al.
Trials, 2018, 19, 1
Cardiac manifestations of congenital LMNA-related muscular dystrophy in children: three case reports and recommendations for care
Heller F, Dabaj I, Mah JK, et al.
Cardiology in the young, 2017, 27, 6, p 1076
Developing standardized corticosteroid treatment for Duchenne muscular dystrophy
Guglieri M, Bushby K, McDermott MP, et al.
Contemporary clinical trials, 2017, 58, p 34
Myostatin inhibitor ACE-031 treatment of ambulatory boys with Duchenne muscular dystrophy: Results of a randomized, placebo-controlled clinical trial
Campbell C, McMillan HJ, Mah JK, et al.
Muscle & Nerve, 2017, 55, 4, p 458
Early onset facioscapulohumeral dystrophy - a systematic review using individual patient data
Goselink RJM, Voermans NC, Okkersen K, et al.
Neuromuscular disorders : NMD, 2017, 27, 12, p 1077
Facioscapulohumeral dystrophy in children: design of a prospective, observational study on natural history, predictors and clinical impact (iFocus FSHD)
Goselink RJ, Schreuder TH, Mul K, et al.
BMC neurology, 2016, 16, 138
Clinical practice considerations in facioscapulohumeral muscular dystrophy Sydney, Australia 21 September 2015
Tawil R, Mah JK, Baker S, et al.
Neuromuscular disorders : NMD, 2016, 26, 7, p 462
Serum pharmacodynamic biomarkers for chronic corticosteroid treatment of children
Hathout Y, Conklin LS, Seol H, et al.
Scientific Reports, 2016, 6
A Systematic Review and Meta-analysis on the Epidemiology of the Muscular Dystrophies
Mah JK, Korngut L, Fiest KM, et al.
The Canadian journal of neurological sciences. Le journal canadien des sciences neurologiques, 2016, 43, 1, p 163
