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RARE Stocks Jump 13% as FDA Approves First Sanfilippo Gene Therapy

Ultragenyx Pharmaceutical Inc. (RARE) received U.S. Food and Drug Administration approval on Thursday for Fayuvi, the first gene therapy for Sanfilippo syndrome type A in pediatric patients. The approval followed a 2025 rejection over manufacturing issues that required the company to resubmit its ap
GateMacroTradfiHub·09-17 22:51
RARE Stocks Jump 13% as FDA Approves First Sanfilippo Gene Therapy