Story thread · 8 reports / 6 sources

FDA Approves First Gene Therapy for Pediatric Patients with Sanfilippo Syndrome Type A

fda.gov · 3h · first report

How the coverage leans

Across 6 sources · syndicated copies counted once

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.

The coverage

How This Gene Therapy Name Partially Reversed Its Catastrophic 44% Dive
investors.com · 50m
STAT+: FDA approves a new gene therapy for Sanfilippo syndrome, an ultra-rare disease
statnews.com · 1h
FDA approves Ultragenyx’s Fayuvi for Sanfilippo syndrome Type A
investing.com · 1h

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