Glycogen Storage Diseases (GSDs) are a group of inherited metabolic disorders that result from deficiencies in enzymes responsible for the synthesis or breakdown of glycogen, a stored form of glucose.

Total Revenue of $560 million, exceeding guidanceCrysvita® revenue of $410 million and Dojolvi® revenue of $88 million2025 Financial ...

Reports Q4 revenue $164.88M, consensus $158.71M. “We have created a next-generation rare disease company on a pathway to profitability with ...

Good afternoon, and welcome to the Ultragenyx Fourth Quarter and Full Year 2024 Financial Results Conference Call. At this time, all participants are in a listen-only-mode. At the end of the prepared ...

A Philadelphia biotechnology company founded seven years ago by two former Johnson & Johnson executives has a new leader.

Ultragenyx Pharmaceutical RARE reported fourth-quarter 2024 loss of $1.39 per share, wider than the Zacks Consensus Estimate ...

The company has completed a Phase 3 study around its candidate DTX401 to treat Glycogen storage disease type I or GSD1a. This is a rare, inherited disorder that inhibits the proper breakdown of ...

Though she acknowledges that there’s ‘still a lot of research to be done’, her efforts to decipher the findings of existing ...

Blue Trust Inc. increased its position in shares of Beam Therapeutics Inc. (NASDAQ:BEAM – Free Report) by 36.3% during the ...

Essential for runners, protein helps your muscles to recover and repair after each workout – but can a protein-packed diet ...