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FDA Approves First Gene Therapy for Sanfilippo Syndrome Type A

Just now September 23, 2026 1 min read 📰 Medical Xpress
📋 Key Takeaway

The FDA approved FAYUVI, the first gene therapy for Sanfilippo syndrome type A, developed by the Abigail Wexner Research Institute at Nationwide Children's Hospital. This approval marks a significant advancement in gene therapy for rare diseases. While the article does not directly relate to Iran, advancements in medical research could influence healthcare policies and gene therapy research in the region.

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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