The FDA cleared Ultragenyx’ one-time AAV8 gene therapy Genglycos (pariglasgene brecaparvovec; DTX-401) for glycogen storage disease type Ia (GSDIa) in patients 8 years and older, marking the first FDA-approved treatment for the underlying enzymatic defect. Separately, Regeneron won FDA approval for Pasatru (garetosmab) to treat fibrodysplasia ossificans progressiva, including reductions in new abnormal bone formation and flare-ups assessed by clinicians.