Sarepta's stock falls 48% after FDA critic Vinay Prasad's appointment and poor Elevidys sales. Company cuts revenue forecast ...

Vertex paused an early-stage trial of its inhaled treatment for cystic fibrosis that came from a partnership with Moderna.

Attorneys general from 19 states and Washington, DC have filed a lawsuit against the federal government alleging that its ...

The FDA on Tuesday said it is planning on doing more surprise inspections of foreign factories, a day after Trump signed an ...

Aldeyra Therapeutics reports Phase 3 success with reproxalap for dry eye disease, plans third FDA filing after previous ...

The European Union is committing €500 million ($556 million) over the next two years to make Europe a “magnet” for ...

Vertex said it's gathering momentum with its new product launches, building out new franchises with its sickle cell therapy ...

A Singapore-based startup raised a $45 million Series B to start testing a gene therapy in humans next year for a genetic ...

Several members of an FDA advisory committee warned on Monday that new data from studies on long-term use of opioids might ...

Function Health is buying Ezra in a bid to create a haven for people who want copious amounts of data on their bodies. The ...

Initial clinical data from Genocury and EsoBiotec shows promise for in vivo CAR-T therapy, with complete remission in ...

PTC Therapeutics announced Monday that its splicing drug for Huntington’s disease met the primary endpoint of a Phase 2 study ...