Exploratory Phase 2a study showed consistent and clinically meaningful functional improvements across muscle strength, motor performance, and ...

Adeno-associated virus (AAV)–mediated gene therapy was one of the focal points of this year's Muscular Dystrophy Association conference. Here, Barry Byrne, MD, PhD, University of Florida, speaks to ...

A phase 2a trial of NMD Pharma’s rare disease prospect ignaseclant has missed its primary endpoint. | A phase 2a trial of NMD ...

Researchers from Carnegie Mellon University have discovered a way to target RNA that could lead to new treatment options for myotonic dystrophy type 1 (DM1), the most common adult-onset form of ...

Neuromuscular diseases are caused by problems in the way muscle cells, motor neurons, and peripheral cells interact. Researchers from the Max Delbrück Center for Molecular Medicine in the Helmholtz ...

Researchers at the UCLA stem cell center and the departments of chemistry and biochemistry and pathology and laboratory medicine have identified, for the first time, a generic way to correct mutations ...

Muscular Dystrophy Association Marks Rare Disease Day by Spotlighting Community Stories Driving Progress in Research, Care, ...

Please provide your email address to receive an email when new articles are posted on . Fitness rates were 10% higher for the exercise group. The effects remained significant over 1 year, but slightly ...

"Muscular dystrophy can take many things from me, but it will not take away my happiness, nor will it take away my faith." ...