Ultragenyx’s GENGLYCOS Becomes First FDA-Approved Gene Therapy for Glycogen Storage Disease Type Ia

Ultragenyx’s GENGLYCOS receives accelerated FDA approval as the first gene therapy targeting the root cause of GSDIa in patients aged 8 and older.

Ultragenyx’s GENGLYCOS Becomes First FDA-Approved Gene Therapy for Glycogen Storage Disease Type Ia
Credit: Ultragenyx Pharmaceutical
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