Background: Casimersen is FDA-approved for treatment of Duchenne muscular dystrophy in patients with exon 45 skip-amenable mutations. ESSENCE is an ongoing, double-blind, placebo-controlled, Phase 3 […]
Background: Delandistrogene moxeparvovec (SRP-9001) is an investigational gene transfer therapy developed for targeted skeletal and cardiac muscle expression of micro-dystrophin, a shortened, functional, dystrophin protein. […]
Background: Delandistrogene moxeparvovec (SRP-9001) is an investigational gene transfer therapy developed for targeted skeletal and cardiac muscle expression of micro-dystrophin, a shortened, functional dystrophin protein. […]
Introduction: Progressive degeneration of muscle cells leading to the loss of ambulation and cardiopulmonary compromise are primary features of Duchenne muscular dystrophy (DMD). In-clinic functional […]
Background: Duchenne muscular dystrophy (DMD) is caused by mutations in the DMD gene resulting in low levels of dystrophin protein, which protects muscle from damage […]
Background: Dystrophinopathy patients often exhibit a range of neuropsychiatric challenges that includes increased risk for anxiety, depression, obsessive-compulsive disorder, ADHD, and externalizing behavioral challenges. Routine […]
Background and Objective Friedreich’s ataxia (FA) is a rare, degenerative neuromuscular disease with no available therapies. Omaveloxolone (Omav) is an investigational drug that activates the […]
Background: Measures of strength and functional abilities may serve as important endpoints for FSHD clinical trials. Objective: To examine the relationship of strength to functional […]
Background Remote pulmonary function testing with improved patient engagement is necessary for clinical decision-making in ALS (Geronimo 2019). Vital capacity stratification may allow identification of […]