FDA approves Ultragenyx's gene therapy for Sanfilippo syndrome type A
The FDA granted full approval to a rare disease gene therapy from Ultragenyx on Thursday, giving the company a much-needed boost in an otherwise difficult year. The therapy, called UX111, will be branded as Fayuvi ...
September 17, 2026 03:04 PM EDT PharmaCell/Gene TxFDA+ FDA approves Ultragenyx's gene therapy for Sanfilippo syndrome type A Max Gelman Senior Editor The FDA granted accelerated approval to a rare disease gene therapy from Ultragenyx on Thursday, giving the company a much-needed boost in an otherwise difficult … Sign up to read this article for free. Get free access to a limited number of articles, plus choose newsletters to get straight to your inbox. SIGN UP LOG IN BECOME A PREMIUM SUBSCRIBER
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