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 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 ...
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Ultragenyx completes BLA filing for GSDIa gene therapy
Ultragenyx Pharmaceutical ( ($RARE) ) just unveiled an update. On December 30, 2025, Ultragenyx Pharmaceutical announced it had completed the ...
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 ...
Ultragenyx Pharmaceutical (RARE) has completed the rolling submission of its U.S. marketing application for its gene therapy ...
If approved, DTX401 will be the first therapy approved to treat the underlying cause of GSDIaNOVATO, Calif., Dec. 30, 2025 ...
US biotech Ultragenyx Pharmaceutical In this week revealed that it has completed the rolling submission of its Biologics ...
The firm submitted non-clinical and clinical modules of its rolling BLA for DTX401 in August and has now submitted the chemistry, manufacturing, and controls module.
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