FDA Approves First Therapy for Patients aged 8 years and older with Glycogen Storage ...

The FDA granted accelerated approval to Genglycos, the first gene therapy for glycogen storage disease type Ia in patients aged 8 and older, based on its ability to reduce cornstarch intake as a surrogate marker for clinical benefit. The approval requires confirmatory trials and positions gene therapy as a treatment option that addresses the root cause of this rare genetic disorder.

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