The FDA is allowing Sarepta to resume shipments of Elevidys (delandistrogene moxeparvovec) to ambulatory patients with Duchenne muscular dystrophy.

Catalyst Pharmaceuticals CPRX is scheduled to report its second-quarter 2025 earnings results on Aug. 6, after market close.

A 12-year-old boy from Carthage, Tennessee finds joy in his power wheelchair. His family are hoping for a cure for his muscular disease, Duchenne.

Deklan Locke, whose nickname is Flash, has a rare illness, Duchenne muscular dystrophy, which causes progressive muscle ...

A cell therapy preserves muscle structure and function in laboratory mice with a type of disease similar to Duchenne muscular ...

Precision BioSciences Inc., a clinical-stage company developing gene editing therapies for diseases with high unmet need, has ...

Dyne Therapeutics' DYNE-251 receives FDA breakthrough status for Duchenne muscular dystrophy treatment. Read more here.

Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage company focused on delivering functional improvement for people living with genetically driven neuromuscular diseases, today announced that the ...

Sarepta and Capricor learned of key regulatory decisions from the media and investors, and Duchenne muscular dystrophy ...

The primary safety risks associated with gene therapy include immune reactions and off-target effects in unintended organs, ...

An alternative to using standard glucocorticoids is the synthetic dissociative steroid vamorolone (Agamree), which was ...

Targeted rewriting of the epigenome promises to overcome several challenges in direct gene editing in patient therapies.