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Ultragenyx Jumps After FDA Approval for $2.7M Gene Therapy

Ultragenyx Pharmaceutical logoUltragenyx Pharmaceutical
Sig3.40Sen+7.10Env0.00Soc+4.60Gov0.00

Ultragenyx’s one-time gene therapy becomes the first approved treatment targeting the underlying cause of GSDIa. The approval follows trial results showing a 61% mean reduction from baseline at Week 96, and a 30 to 60 day path to qualified treatment centers. The U.S. list price is about $2.7 million per patient for an estimated 1,500 to 2,500 Americans.

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