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Ultragenyx Gene Therapy Becomes First FDA-Approved Treatment for Ultra-Rare Disease

1 day ago 7

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Ultragenyx Pharmaceutical’s Genglycos received accelerated FDA approval for glycogen storage disease type Ia, an inherited enzyme deficiency. It’s also the first approved gene therapy for Ultragenyx, which specializes in rare disease treatments.

The post Ultragenyx Gene Therapy Becomes First FDA-Approved Treatment for Ultra-Rare Disease appeared first on MedCity News.

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