Ultragenyx Gene Therapy Is First Approved Treatment for Rare Neuro Disease Sanfilippo

T2Covered by 1 source · first reported by Fierce Biotech / Fierce Healthcare / MedCity / GEN at 18 Sept, 19:38 UTC

Ultragenyx Pharmaceutical’s Fayuvi is now the first FDA-approved therapy for the underlying cause of Sanfilippo syndrome type A, an ultra-rare inherited neurological disorder. It’s the second approved gene therapy for Ultragenyx, which specializes in rare disease medicines. The post Ultragenyx Gene…

science

Sources

• T2Ultragenyx Gene Therapy Is First Approved Treatment for Rare Neuro Disease SanfilippoFierce Biotech / Fierce Healthcare / MedCity / GEN18 Sept, 19:38