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Researchers at Audentes Therapeutics, an Astellas company, are seeking individuals living with late-onset Pompe disease (LOPD) to participate in a phase 1/2 study to confirm safety and efficacy of the investigational…

Lily Ruta shows off the nebula jar craft she made during virtual MDA Summer Camp. Last summer, MDA brought the magic of Summer Camp to kids in the comfort of…

Researchers at Mitsubishi Tanabe Pharmaceutical America (MTPA) are seeking individuals living with amyotrophic lateral sclerosis (ALS) to participate in a six-month observational study. This study is designed to identify biomarkers to serve…

Vaccines recommended by the CDC, including new COVID-19 vaccines, are safe for those with neuromuscular disease. Here’s what you need to know.

Researchers at Ra Pharmaceuticals (now part of UCB BioSciences) are seeking individuals living with generalized myasthenia gravis (gMG) to participate in a phase 3 study to confirm safety, tolerability, and efficacy…

On Feb. 25, the US Food and Drug Administration (FDA) granted accelerated approval to casimersen (Amondys 45) for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to skipping exon…

Researchers at UCB BioSciences are seeking individuals living with generalized myasthenia gravis (gMG) to participate in a phase 3 study. The objective of the study is to confirm the clinical efficacy…

Financial tools and savings options help individuals and families living with neuromuscular disease achieve their goals

A Q&A with Mark Rich, MD, PhD

Mindy Henderson Our biggest challenges can be our greatest advantages