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BioWorld - Tuesday, February 3, 2026
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Home » Ultragenyx fixed on Sanfilippo foe, new data strong
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Ultragenyx fixed on Sanfilippo foe, new data strong

Feb. 3, 2026
By Randy Osborne
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Another data cut from Ultragenyx Pharmaceutical Inc. regarding UX-111 (rebisufligene etisparvovec), an AAV9 gene therapy for type A Sanfilippo syndrome, continued to brighten the picture for the compound, recently taken under review again by the U.S. FDA.
BioWorld Clinical Endocrine/metabolic Gene therapy U.S.

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