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

Abeona Therapeutics® Congratulates Ultragenyx on U.S. FDA Approval of FAYUVI™ Gene Therapy for ...

FDA approval represents a monumental milestone for patients with MPS IIIA and validates Abeona’s foundational R&D and clinical development efforts

Virus To Deliver Working Gene: How Fayuvi Targets Rare Childhood Disease

The U.S. Food and Drug Administration (FDA) has granted approval for Fayuvi, a novel gene therapy developed by Ultragenyx Pharmaceutical Inc. designed to combat Sanfilippo syndrome type A, a devastating rare neurological disorder. A Devastating Childhood Disease Sanfilippo syndrome type A, also known as mucopolysaccharidosis type III A (MPS IIIA), is a progressive and fatal inherited metabolic disease. Children born with this condition lack the …

How This Biotech Partially Reversed Its Catastrophic 44% Dive

Ultragenyx Pharmaceuticals won approval for its newest gene therapy on Thursday, helping shares partially reverse a catastrophic dive.

FDA approves a new gene therapy for Sanfilippo syndrome, an ultra-rare disease

Fayuvi, which was developed by Ultragenyx, is the first treatment for the condition, sometimes known as childhood Alzheimer’s.

Ultragenyx wins FDA approval for pediatric gene therapy for Sanfilippo syndrome

FDA approves Ultragenyx’s Fayuvi, first pediatric gene therapy for Sanfilippo A targeting root cause. Read more here.

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

Silver Spring, Md., Sept. 17, 2026 (GLOBE NEWSWIRE) -- 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., MPS…