As can be seen in the chart above, the company also has ... a Phase 3 study around its candidate DTX401 to treat Glycogen storage disease type I or GSD1a. This is a rare, inherited disorder ...

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.

Glycogen storage disease type IV (GSD IV), caused by a mutation in the glycogen branching enzyme 1 (GBE1) gene, is a rare metabolic disorder with an autosomal recessive inheritance that involves the ...

The predominant inheritance pattern is autosomal recessive except for Anderson-Fabry disease, glycogen storage disease (GSD) type IIb (Danon disease) and mucopolysaccharidosis (MPS) type II (Hunter ...

Besides BEAM-101, BEAM is also expanding its genetic disease pipeline by developing BEAM-301 and BEAM-302 for treating glycogen storage disease type 1a (GSD1a) and alpha-1 antitrypsin deficiency ...

RARE reports mixed fourth-quarter results, as earnings miss estimates while revenues beat the same. Product sales increase year over year.

The disease mainly affects children under 5 years old, but anyone who is unvaccinated is susceptible if exposed. In the 19th and 20th centuries, frequent polio outbreaks made it one of the most ...

However, eating too many carbs without burning them off as energy can lead to fat storage. Finding low carb ... The following chart provides some low carb snack ideas. Low carb snacks can be ...

June 10, 2024 — Scientists have grown 'mini-guts' in the lab to help understand Crohn's disease, showing that 'switches' that modify DNA in gut cells play an ... Major Cause of Inflammatory ...

The Official UK Singles Chart reflects the UK’s biggest songs of the week, based on audio and video streams, downloads, CDs and vinyl, compiled by the Official Charts Company. The UK Top 40 is ...

Ultragenyx Pharmaceutical RARE reported new findings from an ongoing, pivotal study of its investigational candidate, UX111 (ABO-102) AAV gene therapy, for Sanfilippo syndrome type A (MPS IIIA). Per ...

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