Duchenne muscular dystrophy families are sharing stories about Elevidys complications on social media. But they aren’t ...

The death of a boy with Duchenne muscular dystrophy, tied to a gene therapy, is above all a tragedy. But it is also ...

A research team at the University of Osaka has unveiled the molecular mechanism behind genome ejection from adeno-associated ...

CRISPR Therapeutics’ CRSP first marketed product is the one-shot gene therapy Casgevy, approved in late 2023 and early 2024 across the United States and Europe for two blood disorder indications — ...

The clearance ushers in a closely-watched launch for Gilead, which aims to show a long-acting injection can upend a market ...