Shots:
Ultragenyx has completed rolling submission of a BLA to the FDA seeking approval of DTX401 (pariglasgene brecaparvovec) for the treatment of GSDIa
BLA is supported by data from 52 pts with 6yrs. of follow-up. Previously reported P-III (GlucoGene) trial results showed significant reductions in both quantity & frequency of daily cornstarch intake, improved euglycemia…
