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 ...

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

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

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

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

Phase 3 Aspire study enrollment on track to complete in 2025 Aurora study to evaluate GTX-102 in other Angelman syndrome genotypes and ages expected to initiate later this year NOVATO, Calif., June 27 ...

For $45 million upfront, Vor Bio is gaining ex-China rights to RemeGen’s telitacicept, a first-in-class recombinant ...

A new gene therapy delivery device could let hospital pharmacies make personalized nanomedicines to order. This democratized ...

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

Only five days after FDA approval of Yutrepia, Liquidia announced its first commercial shipment of the therapy, making prescriptions available to patients at specialty pharmacies. “We have moved with ...

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