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Gene Therapy

Biotech

🔥 Ultragenyx's First Gene Therapy Costs $2.7 Million. It Buys GSDIa Patients Freedom From the Cornstarch Clock.

The FDA cleared GENGLYCOS, Ultragenyx's first gene therapy: a one-time $2.7 million infusion targeting the root cause of glycogen storage disease type Ia.

Biotech

🔥 Ultragenyx Won Its First Gene Therapy Approval — and the Headline Number Is 31% Less Cornstarch

The FDA approved GENGLYCOS, Ultragenyx's first gene therapy and the first treatment for the root cause of glycogen storage disease type Ia. The headline result: 31% less daily cornstarch.