Bristol Myers Squibb has announced that the FDA has given approval to label updates for Breyanzi and Abecma to treat multiple ...

Viatris’ presbyopia drug MR-141 (phentolamine ophthalmic solution 0.75%) has met the primary endpoint and all secondary ...

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 ...

Cedars-Sinai investigators have developed an investigational therapy that brought a significant number of patients with ...

The Polish government’s new draft law on drug reimbursement aims to streamline procedures, empower patients, and ensure faster and more transparent access to essential therapies.

Investing.com -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) stock gained 2.2% premarket after the company announced that its experimental treatment for Angelman syndrome received Breakthrough ...

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

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

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

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 ...

The FDA has granted Orphan Drug designation to riliprubart for the investigational treatment of AMR in solid organ transplantation.