K. Selby

A randomized placebo-controlled phase 3 trial of an antisense oligonucleotide, drisapersen, in Duchenne muscular dystrophy

J. J. Padilla, C. Palermo, M. Pane, 2018, Neuromuscular Disorders.

Response to the Canadian Agency for Drugs and Technologies in Health and Institut national d’excellence en santé et en services sociaux decision regarding nusinersen for Spinal Muscular Atrophy

B. Brais, A. MacKenzie, C. Campbell, 2018, Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques.

Clinical delineation of the PACS1‐related syndrome—Report on 19 patients

M. Hurles, J. Schuurs-Hoeijmakers, B. D. de Vries, 2016, American journal of medical genetics. Part A.

Symptomatic dystrophinopathies in female children

C. Campbell, D. Biggar, H. Kolski, 2011, Neuromuscular Disorders.

Atypical cerebral palsy: genomics analysis enables precision medicine

C. Ross, W. Wasserman, H. Goez, 2018, Genetics in Medicine.

Towards regulatory endorsement of drug development tools to promote the application of model-informed drug development in Duchenne muscular dystrophy

R. Finkel, A. Pestronk, E. Mazzone, 2019, Journal of Pharmacokinetics and Pharmacodynamics.

Malaysian Siblings with Friedreich Ataxia and Chorea: A Novel Deletion in the Frataxin Gene

T. Snutch, S. Spacey, J. Hukin, 2004, Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques.

A National Spinal Muscular Atrophy Registry for Real-World Evidence

A. McCormick, A. Izenberg, B. Brais, 2020, Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques.

A randomized placebo-controlled phase 3 trial of an antisense oligonucleotide, drisapersen, in Duchenne muscular dystrophy

C. Palermo, M. Pane, E. Mercuri, 2018, Neuromuscular Disorders.