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: Regeneron wins FDA approval to challenge Ipsen for rare bone disease marketNext: Ultragenyx, after setbacks, nabs first gene therapy approval Related Stories Regulatory Bayer’s Hyrnuo set for the NSCLC frontlines on accelerated FDA label expansion pharminent September 10, 2026 Regulatory HHS appoints permanent FDA CBER, CDER heads pharminent September 10, 2026 Regulatory FDA approves AstraZeneca’s Etcamah combo for advanced breast cancer pharminent September 7, 2026