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 ...

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 ...

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 ...

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.

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.

Advocates for veterans are championing Assembly Bill 1103, which would fast-track the approval of academic studies on ...

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

Good morning! Today, we get deeper into the ousting of Nicole Verdun at the FDA, see promise from Vertex’s cell therapy for ...

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

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