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FDA approves first gene therapy for Sanfilippo syndrome, a rare childhood neurodegenerative disease

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Source: STAT
FDA approves first gene therapy for Sanfilippo syndrome, a rare childhood neurodegenerative disease
Photo: STAT
TL;DR

The FDA approved Ultragenyx’s gene therapy Fayuvi as the first treatment for Sanfilippo syndrome type A, a rare pediatric neurodegenerative disease, with price not disclosed.

Topics:businesshealth#fayuvi#fda-approval#gene-therapy#health#sanfilippo-syndrome#ultragenyx
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  1. FDA approves a new gene therapy for Sanfilippo syndrome, an ultra-rare disease  STAT
  2. FDA approves Ultragenyx’s gene therapy Fayuvi for rare neurodegenerative disorder  Fierce Pharma
  3. Ultragenyx Gets A ‘Much-Needed Win,’ Says Cantor – Sees $400M Opportunity After FDA Approves Neurodegenerative Disease Therapy  Yahoo Finance
  4. Ultragenyx sets $3.95 million price for rare disease gene therapy  Reuters
  5. Updated: Ultragenyx’s new gene therapy will cost $3.95 million  Endpoints News

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