The interim data found one child in the Duchenne trial produced more than double the amount of the protein needed to maintain ...

Researchers combined advanced imaging techniques and theoretical physics to observe and explain how nanoclusters of the protein emerin form inside living cells. The study uncovers the molecular 'rules ...

RGX-202, a DMD gene therapy, has been well tolerated in an ongoing clinical trial and improved motor function for DMD boys, ...

A patient has died while taking a closely watched gene therapy for muscular dystrophy. Sarepta Therapeutics announced the ...

Preservation of Skeletal Muscle Function Shown Over 3 Years Resulting in 52% Slowing of Disease-- --Data Presented at the ...

Regenxbio's RGX-202 gene therapy shows promising results in Duchenne muscular dystrophy, with high microdystrophin levels and ...

Myotonic dystrophy type 1 (DM1) is the most common adult-onset form of muscular dystrophy and a condition that severely ...

Muscular Dystrophy Association Clinical & Scientific Conference, convening in Dallas, Texas, from March 16-18, will feature ...

A young man treated with Elevidys died of acute liver failure. The case may give doctors pause before prescribing the ...

A young man with Duchenne MD and treated with Elevidys has died of acute liver failure, Sarepta reports; it's working with ...

Giulio Cossu, MD, speaks to the lingering safety concerns related to ex vivo gene therapy in Duchenne muscular dystrophy (DMD) as long-term data are yet to be established.

Exploring Oviedo in Results-1 WASHINGTON – Sarepta Therapeutics said Tuesday that a patient died while taking its closely watched gene therapy for muscular dystrophy, sending company shares ...