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medicalxpress.com · Sep 23

FDA approves first gene therapy treatment for Sanfilippo syndrome type A

The Abigail Wexner Research Institute at Nationwide Children's Hospital celebrated the Food and Drug Administration's (FDA) approval of FAYUVI for Sanfilippo syndrome type A, following years of research in its Jerry R. Mendell Center for Gene Therapy to help patients with devastating diseases.

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