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glycogen storage disease type ia with a 17 year history of renal involvement a case report
Original title: “Glycogen storage disease type Ia with a 17-year history of renal involvement: a case report.”
PDUFA action date set for August 23, 2026 If approved, DTX401 will be the first treatment to address the underlying cause of GDSIa NOVATO, Calif., Feb. ... The company has built a diverse portfolio of approved medicines and treatment candidates aimed at addressing diseases with high unmet medical need and clear biology, for which there are typically no approved therapies treating the underlying disease. The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. About Ultragenyx Ultragenyx is a biopharmaceutical company committed to bringing novel therapies to patients for the treatment of serious rare and ultra-rare genetic diseases. (NASDAQ: RARE) today announced the U.S. Food and Drug Administration (FDA or the Agency) has accepted for review the Biologics License Application (BLA) seeking approval of DTX401 AAV gene therapy (pariglasgene brecaparvovec) for the treatment of Glycogen Storage Disease Type Ia (GSDIa). Such risks and uncertainties include, among others, the uncertainty of clinical drug development and unpredictability and lengthy process for obtaining regulatory approvals, the ability of the company to successfully develop DTX401, the company’s ability to achieve its projected development goals in its expected timeframes, risks related to adverse side effects, risks related to reliance on third party partners to conduct certain activities on the company’s behalf, the company’s limited experience in operating its own manufacturing facility, the ability of the company and its third party
Original title: “Ultragenyx Announces U.S. FDA Acceptance and Priority Review of the Biologics License Application (BLA) for DTX401 AAV Gene Therapy for Glycogen Storage Disease Type Ia (GSDIa)”
proteomics profiling of serum and liver in gsd ia and ib patients insights into complication mechanisms and circulation biomarkers
Original title: “Proteomics profiling of serum and liver in GSD ia and Ib patients: insights into complication mechanisms and circulation biomarkers.”