FDA Approves First Gene Therapy for Pediatric Patients with Sanfilippo Syndrome Type A
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What happened
The U.S. Food and Drug Administration today approved Fayuvi (rebisufligene etisparvovec-hopf), the first treatment for pediatric patients with mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo syndrome type A.
Summary assembled by rule from the sources below