FDA approves first gene therapy treatment for Sanfilippo syndrome type A

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FDA approves first gene therapy treatment for Sanfilippo syndrome type A

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The FDA approved FAYUVI, the first gene therapy treatment for Sanfilippo syndrome type A, developed at the Abigail Wexner Research Institute at Nationwide Children's Hospital. This milestone offers new hope for patients suffering from this devastating genetic disorder.

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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