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 ...

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

A new discovery about how tiny protein clusters form in cells could pave the way for treatments for Emery-Dreifuss muscular ...

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

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

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

Precision BioSciences (DTIL) announced the presentation of preclinical data for its PBGENE-DMD development program for the treatment of ...

A new discovery about how tiny protein clusters form in cells could pave the way for treatments for Emery-Dreifuss muscular dystrophy (EDMD), a rare genetic disorder that causes muscle weakness and ...

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

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.

AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced the advancement of the Phase 1/Phase 2 LION-CS101 clinical trial of ...