Background: Vamorolone is a dissociative steroidal anti-inflammatory that seeks to retain efficacy and reduce safety concerns compared to corticosteroids via changes to structure/activity relationships with […]
Objectives: Evaluate long-term safety and efficacy of losmapimod for the treatment of FSHD. Background: FSHD is caused by aberrant expression of DUX4. Losmapimod is an […]
Objective: Evaluate feasibility to monitor daily activity and assess functional outcomes using wearable sensor devices in an open label study (OLS) of losmapimod in facioscapulohumeral […]
Background: Pain is a recognized symptom of muscular dystrophy (MD), but little is known about prescription medication use for MD-related pain management. Using population-based surveillance […]
Background: The phenotype of Becker muscular dystrophy (BMD) is extremely variable, ranging from Duchenne-like severity (loss of ambulation in second decade) to much milder disease […]
Background: Genetic variations in ALS may influence the rate of disease progression, survival, and serve as pharmacogenomic biomarkers. We evaluated the relationship of ALS-linked genes […]
Introduction: Myotonic dystrophy type 1 (DM1) is a rare neuromuscular disease caused by the expansion of CUG repeats in the 3’-untranslated region of the dystrophia […]
Spinal muscular atrophy (SMA) is a neuromuscular disorder caused my mutation of the Survival Motor Neuron 1 (SMN1). SMA is characterized by degeneration of motor […]
Background: Spinal Muscular Atrophy (SMA) is a genetic disease that is caused by a mutation in the SMN1 gene that greatly reduces the synthesis of […]