The president and CEO of the Muscular Dystrophy Association talks about what she's most excited about in neuromuscular disease research.

UniQure and REGENXBIO are both dealing with FDA setbacks for their respective gene therapies, as regulatory experts question the FDA’s decision-making processes; CBER director Vinay Prasad is under ...

When Ingram became Sarepta Therapeutics’ CEO in 2017, he didn’t have a connection to muscular dystrophy, but he has developed ...

Researchers at the University of Basel have developed a gene therapy that could potentially treat a rare and currently fatal ...

Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...

Panelists discuss how recent advancements in muscular dystrophy treatment have evolved toward truly disease-modifying therapies using gene replacement, antisense oligonucleotides, and gene transfer ...

We hypothesize that Duchenne muscular dystrophy and autism spectrum disorder/pervasive developmental disorder co-occur with a greater than random frequency. In this study, we set out to reject the ...

Physical activity and rehabilitation are key components of Duchenne muscular dystrophy treatment, despite recent drug therapy advances. "Rehabilitation care and rehabilitation teams with experience ...

Congenital muscular dystrophy (CMD) is a disease that affects certain muscles. Individuals with CMD may experience symptoms such as muscle weakness and joint problems. “Congenital” means present since ...

Sen. Roger Wicker urges states to add Duchenne muscular dystrophy to newborn screening, after federal health officials back ...