A few short days after announcing an FDA pivot on a separate asset, REGENXBIO is planning to test the agency’s apparent ...

Scientists have created a new gene therapy for Duchenne muscular dystrophy (DMD) that may not only help stop the disease in DMD patients, but might also help restore their damaged muscles in the ...

Emma Ciafaloni, MD, FAAN, dives into the latest developments in gene therapy for muscular dystrophies, focusing on Duchenne muscular dystrophy, and discusses challenges, genetic causes, and the ...

Regenxbio plans to submit its Duchenne gene therapy for FDA approval, just a month after it said that regulators wanted to ...

Company's Biologics License Application on track with PDUFA target action date of August 22, 2026– SAN DIEGO, June 26, 2026 ...

A new gene therapy treatment for Duchenne muscular dystrophy shows promise of not only arresting the decline of the muscles of those affected by this inherited genetic disease, but perhaps, in the ...

Muscular dystrophy is a disease caused by mutations in the dystrophin gene. The muscles of muscular dystrophy patients weaken and atrophy over time. Heart and breathing muscles may also eventually be ...

A specific gene may play a key role in new treatments that prevent muscle in the body from breaking down in serious muscle diseases, muscular dystrophies. This is shown in a new study at Umeå ...

A major trial of a promising gene therapy for Duchenne muscular dystrophy has fallen short, but researchers say the lessons learned could pave the way for better, more effective treatments. A newpaper ...

The decreasing cost and growing accessibility of next-generation sequencing have facilitated the application of whole-exome sequencing (WES) and whole-genome sequencing (WGS) in clinical genetics.

Myotonic dystrophy is thought to be caused by the binding of a protein called Mbnl1 to abnormal RNA repeats. In these two images of the same muscle precursor cell, the top image shows the location of ...

Learn more Servier has signed an agreement to acquire Edgewise Therapeutics’ muscular dystrophy business in a deal valued at up to $2.65bn. The value includes a $1.55bn upfront payment and as much as ...