Glycogen storage disease (GSD) refers to a group of rare genetic conditions that affect how the body stores and breaks down glycogen. The most common type is type I, also called von Gierke disease.

NOVATO, Calif., July 26, 2018 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare ...

Ultragenyx's AAV8 vector gene therapy, DTX401, is advancing through clinical trials as potentially the first ever therapy to treat Glycogen Storage Disease Type 1a (GSD-1a). NEWTON, Mass., Sept. 28, ...

Glycogen storage diseases (GSDs) comprise a heterogeneous group of inherited metabolic disorders that impede the normal synthesis or degradation of glycogen, resulting in diverse clinical phenotypes.

Treatment with DTX401 resulted in a statistically significant reduction in daily cornstarch intake at Week 48 (p<0.0001) with maintenance of glucose control Company will host investor call today at ...

Single Dose of BEAM-301 Restored Clinically Meaningful Endpoints in In Vivo Rodent Disease Models Out to at Least One Year Company Plans to Submit U.S. Investigational New Drug (IND) Application for ...

NOVATO, Calif., Aug. 18, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced the initiation of a rolling submission of a Biologics License Application (BLA) to the U ...

Pompe disease is a rare genetic condition that affects only 1 person in every 40,000 people in the United States, according to the National Institute of Neurological Disorders and Stroke (NINDS).