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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) ...
Mena FN

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)

(MENAFN- GlobeNewsWire - Nasdaq) If approved, DTX401 will be the first therapy approved to treat the underlying cause of GSDIa NOVATO, Calif., Dec. 30, 2025 (GLOBE NEWSWIRE) -- Ultragenyx ...
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Ultragenyx Pharmaceutical Inc.: Ultragenyx Completes Rolling Submission of Biologics License Application (BLA) to U.S. FDA for DTX401 AAV Gene Therapy for Glycogen Storage ...

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) ...
Medscape

Diagnosis and Management of Benign Liver Tumors

Semin Liver Dis. 2013;33(3):236-247. Focal nodular hyperplasia has no malignant potential and usually remains stable or decreases in size over time. Less than 20% of patients with FNH develop symptoms ...
Medical Xpress

Multiple Sulfatase Deficiency Disease

In two new papers, researchers from The Jackson Laboratory (JAX) report the successful use of two approaches—gene therapy and bone marrow transplantation—to alleviate symptoms of multiple sulfatase ...
Nature

A new avenue for CNS biotherapeutics

JCR Pharmaceuticals’ unique brain-shuttling technology opens new treatment opportunities for meeting the needs of patients grappling with CNS diseases. Getting drugs from the bloodstream into the ...