Shares of Ultragenyx Pharmaceutical RARE were up 15.5% yesterday following the completion of the rolling submission of a ...

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that it has completed the rolling submission of its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA), ...

Ultragenyx (RARE) announced that it has completed the rolling submission of its biologics license application to the FDA, seeking approval for ...

The top 10 PharmTech videos of the year cover advanced therapies, high-concentration biologics, major mergers and ...

All in all, PHAT has 6 recent analyst reviews that break down into 5 Buys and 1 Hold, giving the stock a Strong Buy consensus rating. The shares are priced at $17.66 and have an average price target ...

GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative ...

Israel's Ministry of Health authorizes individual patients early access treatment with GS010/LUMEVOQ, a candidate gene therapy for the treatment of ND4-LHON. Bilateral injection expected to ...

Findings from two clinical trials suggest Novartis' Itvisma is safe and effective for SMA patients over 2 years old.

Revolutions in gene therapy are rapidly changing the landscape of modern medicine. Revolutions in gene therapy are rapidly changing the landscape of modern medicine, forcing society and science alike ...

Novartis has received an FDA approval for Itvisma, a new version of the company’s gene therapy Zolgensma, to treat older patients with spinal muscular atrophy (SMA). The approval comes more than six ...

A California toddler is the first person in the world to receive gene therapy to treat his devastating disease. Three-year-old Oliver Chu was born with a rare, genetic condition called Hunter syndrome ...

WATERTOWN, Mass.--(BUSINESS WIRE)--Dyno Therapeutics, Inc., a genetic technologies company applying artificial intelligence (AI) to solve the grand challenge of in vivo gene delivery, announced ...