If a deal is confirmed and consummated, Novartis would claim ownership of a biotech that has raised something of a stir in ...

After a second Elevidys patient death from acute liver failure, Sarepta in July launched a major restructuring that involves ...

Avidity Biosciences stock rocketed Wednesday on a report Novartis made an attempt to buy the rare disease-focused biotech ...

Keros Therapeutics, Inc. (“Keros”) (Nasdaq: KROS), a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapeutics to treat a wide range of patients with ...

Sarepta Therapeutics hired a Trump-connected lobbying firm after the death of a teenage boy treated with its Duchenne ...

Dr Vinay Prasad was asked to step down as the FDA's top vaccine official following several controversies during his brief tenure.

Cumberland Pharmaceuticals' Q2 2025 earnings highlight revenue growth, clinical progress in ifetroban for DMD, and Vibativ's global expansion.

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

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 ...