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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 Therapy Is First Approved Treatment for Rare Neuro Disease Sanfilippo appeared first on MedCity News.
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