Sage Journals: Discover world-class research

Open access

Meeting report

First published 2024-03-05

Draft Guidance for Industry Duchenne Muscular Dystrophy,Becker Muscular Dystrophy,and Related Dystrophinopathies – Developing Potential Treatments for the Entire Spectrum of Disease

Craig McDonald, Eric Camino, Rafael Escandon [...]

Journal of Neuromuscular Diseases

Open access

Research article

First published 2021

Open-Label Evaluation of Eteplirsen in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping: PROMOVI Trial

Craig M. McDonald, Perry B. Shieh, Hoda Z. Abdel-Hamid [...]

Journal of Neuromuscular Diseases

Free access

Research article

First published 2020

Characterizing Enrollment in Observational Studies of Duchenne Muscular Dystrophy by Race and Ethnicity

Alison M. Barnard, Samuel L. Riehl, Rebecca J. Willcocks [...]

Journal of Neuromuscular Diseases

Open access

Review article

First published 2018

“Of Mice and Measures”: A Project to Improve How We Advance Duchenne Muscular Dystrophy Therapies to the Clinic

Heather Gordish-Dressman, Raffaella Willmann, Laura Dalle Pazze [...]

Journal of Neuromuscular Diseases

Open access

Research article

First published 2018-2-21

Long-Term Pulmonary Function in Duchenne Muscular Dystrophy: Comparison of Eteplirsen-Treated Patients to Natural History

T. Bernard Kinane, Oscar H. Mayer, Petra W. Duda [...]

Journal of Neuromuscular Diseases

Retricted access

Research article

First published 2016-11

Immunomodulatory therapies delay disease progression in multiple sclerosis

Roberto Bergamaschi, Silvana Quaglini, Eleonora Tavazzi [...]

Multiple Sclerosis Journal

Retricted access

Research article

First published 2023-05

T Cell Responses to Dystrophin in a Natural History Study of Duchenne Muscular Dystrophy

Karen Anthony, Pierpaolo Ala, Francesco Catapano [...]

Human Gene Therapy

Open access

Research article

First published 2023-05-02

Efficacy and Safety of Viltolarsen in Boys With Duchenne Muscular Dystrophy: Results From the Phase 2,Open-Label,4-Year Extension Study

Paula R. Clemens, Vamshi K. Rao, Anne M. Connolly [...]

Journal of Neuromuscular Diseases

Open access

Research article

First published 2019

Molecular and Clinical Characteristics of a National Cohort of Paediatric Duchenne Muscular Dystrophy Patients in Norway

Ellen Johanne Annexstad, Toril Fagerheim, Inger Holm [...]

Journal of Neuromuscular Diseases

Open access

Review article

First published 2021-07-30

Early Gross Motor Milestones in Duchenne Muscular Dystrophy

Giulia Norcia, Simona Lucibello, Giorgia Coratti [...]

Journal of Neuromuscular Diseases

Search results