FDA Approves First Therapy for Patients aged 8 years and older with Glycogen Storage Disease Type Ia

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Summary

FDA approves the first therapy (Genglycos) for patients aged 8 years and older with glycogen storage disease type Ia (GSDIa) under accelerated approval.

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Diseases / indications

• Glycogen Storage Disease Type Ia

Drugs mentioned

• Genglycos (pariglasgene brecaparvovec-opnr)

Snippet

The U.S. Food and Drug Administration today issued an accelerated approval for Genglycos (pariglasgene brecaparvovec-opnr) as the first-ever treatment for adults and children 8 years of age and older with glycogen storage disease type Ia (GSDIa)