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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 rolling submission of its Biologics License Application to ...
Yahoo Finance

RARE Rises on Completion of Rolling Submission for AAV Gene Therapy

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Yahoo Finance

Beam Therapeutics Announces Preclinical Data Highlighting Base Editing Approach to Correct a Glycogen Storage Disease Type Ia Disease-Causing Mutation

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Business Wire

Dimension Therapeutics to Develop Gene Therapy Treatments for Patients with Ornithine Transcarbamylase (OTC) Deficiency and Glycogen Storage Disease Type Ia (GSDIa) | Business Wire

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Dimension Therapeutics, Inc. (“Dimension” or the “Company”), a rare disease company advancing novel, liver-directed treatments for diverse genetic disorders, today ...
Nasdaq

Ultragenyx Completes FDA BLA Submission For DTX401 Gene Therapy To Treat GSDIa

(RTTNews) - Ultragenyx Pharmaceutical Inc. (RARE) announced that it has completed the rolling submission of its Biologics License Application (BLA) to the U.S. Food and Drug Administration for DTX401 ...
Medical Xpress

Research yields potential treatment approach for glycogen storage disease

Researchers from the Duke-NUS Graduate Medical School Singapore (Duke-NUS) and Duke Medicine have identified a potential treatment strategy for an often-fatal inherited glycogen storage disease.
Taiwan News

Ultragenyx Announces Positive Longer-term Data from Phase 3 Study of DTX401 AAV Gene Therapy for the Treatment of Glycogen Storage Disease Type Ia (GSDIa)

At Week 96 participants experienced even greater reductions in daily cornstarch intake while maintaining low levels of hypoglycemia, improved levels of euglycemia and improved fasting tolerance Phase ...
The Globe and Mail

Ultragenyx Completes Rolling Submission of Biologics License Application (BLA) to U.S. FDA for DTX401 AAV Gene Therapy for Glycogen Storage Disease Type Ia (GSDIa)

NOVATO, Calif., Dec. 30, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that it has completed the rolling submission of its Biologics License Application (BLA) ...
Science Daily

Potential treatment approach for glycogen storage disease

A potential treatment strategy for an often-fatal inherited glycogen storage disease has been identified by researchers. Patients with the disorder lack the enzyme that enables their livers to convert ...
News Medical

Potential treatment strategy for glycogen storage disease

Researchers from the Duke-NUS Graduate Medical School Singapore (Duke-NUS) and Duke Medicine have identified a potential treatment strategy for an often-fatal inherited glycogen storage disease.