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Josh Corbett[Image description: Josh, a light-skinned boy with short brown hair, stands in front of a brick wall wearing a black jacket, gray shirt, red shorts, and blue sneakers.]  …

Researchers at Apellis Pharmaceuticals are seeking individuals living with sporadic amyotrophic lateral sclerosis (ALS) to participate in a two-year phase 2 trial (MERIDIAN) to determine the efficacy of the investigational drug…

When you can’t speak for yourself, these important documents help you protect yourself and your dependents

Researchers at Johns Hopkins University School of Medicine are seeking individuals living with amyotrophic lateral sclerosis (ALS), primary lateral sclerosis (PLS), or a related motor neuron disease to participate in a…

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…