glycogen storage disease type 1

FDA Rejects Ultragenyx’s Gene Therapy for Rare Disease, Citing Manufacturing Concerns

Post author:Bree Clare
Post published:July 25, 2025
Post category:Glycogen Storage Disease Type I

Ultragenyx Pharmaceutical, a biotechnology company developing treatments for rare diseases, has received a setback from the U.S. Food and Drug Administration (FDA) regarding its gene therapy candidate, DTX401. According to…

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New Data Available for GSD1a, OTC Deficiency, and Wilson Disease Studies

Post author:Jessica Lynn
Post published:January 19, 2021
Post category:Glycogen Storage Disease Type I ornithine transcarbamylase deficiency Wilson Disease

In a recent press release, biopharmaceutical company Ultragenyx Pharmaceutical Inc. ("Ultragenyx") shared positive safety and efficacy data from Phase 1/2 clinical trials on gene therapy solutions for ornithine transcarbamylase…

FDA Rejects Ultragenyx’s Gene Therapy for Rare Disease, Citing Manufacturing Concerns

New Data Available for GSD1a, OTC Deficiency, and Wilson Disease Studies

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