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.

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

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

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

In 2024, Ultragenyx announced that the phase III GlucoGene study (NCT05139316), evaluating its investigational AAV8 gene therapy, DTX401, to treat glycogen storage disease type Ia patients aged ...

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 ...

BEAM-301 is also an in vivo gene editing approach, with the intention of targeting glycogen storage disease 1a. They expect to start dosing patients in early 2025. BEAM is also continuing to work ...

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 ...

Storage diseases associated with restrictive cardiomyopathy include Fabry’s disease, Gaucher’s disease, and glycogen storage diseases. 2,3 Although this patient had angiomas on the skin ...