The agency removed some onerous requirements for the complex cancer drugs and reduced restrictions on patients’ ...

Label updates have been approved by the FDA for the CAR T-cell therapies Breyanzi and Abecma in large B cell lymphoma and ...

Ultragenyx Pharmaceutical received breakthrough therapy designation from the U.S. Food and Drug Administration for its drug GTX-102, which is designed to treat people with Angelman syndrome. Angelman ...

In one of the first major tests of the new FDA leadership’s regulatory philosophy toward gene therapies for rare diseases, ...

U.S. Department of Health and Human Services Secretary Robert F. Kennedy Jr. says his agency is “absolutely committed” to ...

Simon Fraser University researchers are using a new approach to brain imaging that could improve how drugs are prescribed to ...

Mary Catchpole, 19, suffers from inherited activated PI3-Kinase delta syndrome (APDS), a life-threatening condition.

The Polish government’s new draft law on drug reimbursement aims to streamline procedures, empower patients, and ensure ...

Shares of Sarepta Therapeutics have plunged again after the drugmaker reported a second death in connection with its gene ...

The U.S. Food and Drug Administration said on Tuesday that it is investigating reports of two deaths due to acute liver ...