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 Scholar Rock’s muscle-targeting SMA therapy Isemblyd lands FDA approval pharminent September 14, 2026 Regulatory Psychedelic therapies ride wave of public favor into FDA hearing pharminent September 14, 2026 Regulatory Bayer’s Hyrnuo set for the NSCLC frontlines on accelerated FDA label expansion pharminent September 10, 2026