Last Updated: 09/26/2026
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Found 103 publications
Cardiotoxic Effects and Microdystrophin Expression after Gene Therapy for DMD.
Journal: The New England journal of medicine
Published: July 22, 2026
Prospective Study of Video Hand Opening Time as a Quantitative Measurement of Myotonia in Patients With Myotonic Dystrophy Type 1.
Journal: Neurology
Published: February 26, 2026
Learnings from Patient Mortality after Delandistrogene Moxeparvovec Administration: A Report of Two Cases and Expert Committee Considerations for Future Mitigation and Management.
Journal: Human gene therapy
Published: February 13, 2026
A randomized, double-blind, placebo-controlled study of losmapimod in patients with facioscapulohumeral muscular dystrophy: Results of the REACH study.
Journal: Journal of neuromuscular diseases
Published: February 06, 2026
Longitudinal Psychometric Properties of the Myotonic Dystrophy Health Index in a Large Multicenter Cohort of People Living With Myotonic Dystrophy Type 1.
Journal: Muscle & nerve
Published: December 05, 2025
Electrical Impedance Myography Detects Disease Progression over 12 to 24 Months in Facioscapulohumeral Muscular Dystrophy.
Journal: Annals of neurology
Published: November 14, 2025
Late-onset facioscapulohumeral muscular dystrophy defines a distinct clinical subgroup.
Journal: Neuromuscular disorders : NMD
Published: November 14, 2025
Trach and treat: Safety and motor outcomes following onasemnogene abeparvovec in patients with spinal muscular atrophy and tracheostomies in the RESTORE registry.
Journal: Journal of neuromuscular diseases
Published: November 11, 2025
Establishing biomarkers and clinical endpoints in myotonic dystrophy type 1 (END-DM1): Protocol of an international natural history study.
Journal: PloS one
Published: September 11, 2025
Consensus recommendations and considerations for the delivery and monitoring of gene therapy in patients with Duchenne muscular dystrophy.
Journal: Neuromuscular disorders : NMD
Published: June 17, 2025
Gene therapy for children with X-linked myotubular myopathy: a plain language summary of publication for the ASPIRO study.
Journal: Therapeutic advances in rare disease
Published: April 08, 2025
Switching to subcutaneous zilucoplan from intravenous complement component 5 inhibitors in generalised myasthenia gravis: a phase IIIb, open-label study.
Journal: Therapeutic advances in neurological disorders
Published: March 04, 2025
Last Updated: 09/26/2026