Ultragenyx Pharmaceutical recently completed its rolling Biologics License Application to the FDA for DTX401 gene therapy for Glycogen Storage Disease Type Ia, while also reporting Phase 3 success for ...

If approved, DTX401 will be the first therapy approved to treat the underlying cause of GSDIaNOVATO, Calif., Dec. 30, 2025 ...

Glycogen storage diseases (GSDs) constitute a group of inherited metabolic disorders that impair normal glycogen synthesis or degradation, resulting in the accumulation of structurally abnormal ...

Ultragenyx Pharmaceutical ( ($RARE) ) just unveiled an update. On December 30, 2025, Ultragenyx Pharmaceutical announced it had completed the ...

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.

When we see an individual with abnormal left ventricular hypertrophy and preexcitation, our first thought should not be HCM, it should be a glycogen-storage disease. The Seidmans are coauthors of the ...

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

At the Association for Glycogen Storage Disease's 41st Annual Conference, Dr. David Weinstein of UConn School of Medicine and Connecticut Children's presented his groundbreaking, one-year clinical ...

This case presents an infant male child who initially presented with clinical features resembling glycogen storage disease type I (GSD I), including hepatomegaly, a doll-like face and metabolic ...

McArdle's disease, also known as Glycogen Storage Disease Type V, or GSD5, is a congenital disorder of muscle metabolism. It occurs when the body loses its ability to break down glycogen leading to ...

Unexplained left ventricular hypertrophy often prompts the diagnosis of hypertrophic cardiomyopathy, a sarcomere-protein gene disorder. Because mutations in the gene for AMP-activated protein kinase γ ...

The rare and deadly genetic liver disorder, GSD type Ia, affects children from infancy through adulthood, causing dangerously low blood sugar levels and constant dependence on glucose consumption in ...