The global medical community is celebrating a massive milestone following the FDA’s historic approval of Fayuvi (rebisufligene etisparvovec-hopf), the first-ever gene therapy designed to treat children suffering from Mucopolysaccharidosis Type IIIA (Sanfilippo syndrome Type A). This progressive and devastating neurodegenerative disease causes children to lose cognitive and language skills over time. By utilizing an adeno-associated virus vector to fix the underlying genetic error, this single-dose intravenous infusion offers a long-awaited lifeline to families who previously had no treatment options beyond basic symptom management.
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