FDA Approves Rebisufligene Etisparvovec as First Gene Therapy for Children With ...

The FDA approved rebisufligene etisparvovec-hopf (Fayuvi), the first gene therapy to treat children with mucopolysaccharidosis type IIIA (Sanfilippo syndrome type A), providing a disease-modifying option. The therapy uses AAV9 to deliver a functional SGSH gene, showing cognitive benefits in young patients versus historical controls, with key safety risks requiring close monitoring.

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