While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several ...

Application is supported by results from the DEVOTE study, which suggested that two doses of Spinraza (nusinerse) 50 mg taken ...

For this study, outcomes were compared between 2 groups of patients with myasthenia gravis: those who developed exacerbations ...

Jonathan Lengel is an actor, singer, and performer who lives with a rare form of congenital muscular dystrophy. He received an inaugural MDA College Scholarship in 2024 to pursue his academic goals in ...

Topline results from a phase 3 clinical trial showed an investigational gene therapy for young people with spinal muscular atrophy type 2 increased mobility and slowed disease progression ...

Riding recent momentum in the Duchenne muscular dystrophy space, Capricor Therapeutics, Wave Life Sciences, Regenxbio and ...

Dyne Therapeutics received fast-track designation from the Food and Drug Administration for a treatment of myotonic dystrophy type 1, a neuromuscular disease.

Neurology is a branch of medicine that focuses on disorders of the nervous system, including the brain, spinal cord, and nerves. Neuromuscular diseases, which affect the muscles and their direct ...

Biogen said the U.S. Food and Drug Administration has accepted its supplemental new-drug application, while the European Medicines Agency has validated its application, which confirms that the ...

By clicking “Accept All Cookies”, you agree to the storing of cookies on your device to enhance site navigation, analyze site ...

Ankle-foot orthoses (AFOs) may reduce the effort required to walk, and increase walking speed and satisfaction with walking in people with calf muscle weakness due to neuromuscular disorders, but we ...

Altruism values for treatments of rare, severe pediatric diseases have not been estimated. This study found the altruism value for a hypothetical new Duchenne muscular dystrophy treatment to be $80 ...