Background and Objective Friedreich’s ataxia (FA) is a rare, degenerative neuromuscular disease with no available therapies. Omaveloxolone (Omav), an investigational drug, is an activator of […]
Background and Objective Friedreich’s ataxia (FA) is a rare, inherited, degenerative, neuromuscular disease. Prevalence estimates in the US indicate ~4000 patients are living with FA. […]
Background and Objectives: The natural history of Friedreich Ataxia (FA) is being investigated in a multi-center longitudinal study designated as the Friedreich Ataxia Clinical Outcome […]
Objective: Evaluate the safety and tolerability of losmapimod in the treatment for FSHD. Background: FSHD is a relentless, variably progressive disease leading to accumulation of […]
Objective: Evaluate muscle ultrasound (US) in an open-label trial of losmapimod in FSHD1 patients. Background: Natural history studies have identified US as a viable imaging […]
Clinical trials of genotype-targeted therapies in DMD typically compare treated patients to genotype-matched controls. While this avoids confounding by genotype effects, the pool of eligible […]
Use of real-world and natural history data (RWD/NHD) as external controls for pulmonary outcomes is of interest for drug development in ambulatory Duchenne muscular dystrophy […]
The North Star Ambulatory Assessment (NSAA), which consists of 17 functional items, is foundational for ambulatory DMD care and is regularly conducted per care guidelines […]
Background: Amyotrophic Lateral Sclerosis (ALS) in a neurodegenerative disease that results in the progressive deterioration and loss of function of the motor neurons leading to […]