Regulatory Ultragenyx wins FDA approval for first treatment targeting root cause of GSDIa PharmaphorumUltragenyx secured its first gene therapy approval, the FDA clearing GENGLYCOS as the first treatment designed to address underlying cause of GSDla.Rare DiseaseRead full story pharminent August 20, 2026 (Last updated: August 21, 2026) Pharmaphorum Ultragenyx secured its first gene therapy approval, the FDA clearing GENGLYCOS as the first treatment designed to address underlying cause of GSDla. Rare Disease Read full story Post navigation Previous: Moderna and Merck’s Personalized Melanoma Vaccine Scores Historic Phase 3 WinNext: Cancer vaccine win restores Wall Street’s faith in Moderna Related Stories Regulatory Trump nominates policy aide Overton to lead FDA pharminent August 19, 2026 Regulatory Lantheus Holdings’ Alzheimer’s radiodiagnostic secures FDA approval pharminent August 17, 2026 Regulatory BMS bags first approval for myeloma CELMoD Zenbexus pharminent August 14, 2026
