Ultragenyx Announces Positive Longer-Term Data Demonstrating Treatment with UX111 Gene Therapy Results in Sustained, Significant Reductions in CSF-HS and Continued Meaningful Improvements ...
Ultragenyx Pharmaceutical Inc. (RARE)
Last ultragenyx pharmaceutical inc. earnings: 2/13 04:01 pm
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Source: GlobeNewswire
Data represents up to 8.5 years of follow-up and are consistent across age, dose, and genotype BLA resubmitted to U.S. FDA in January 2026; Company expects up to six-month review period per FDA guidelines NOVATO, Calif., Feb. 03, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. today announced new long-term data from clinical studies evaluating UX111 (rebisufligene etisparvovec), an investigational AAV9 gene therapy for Sanfilippo syndrome Type A (MPS IIIA), a fatal neurodegenerative lysosomal storage disorder. The results demonstrate substantial and durable biomarker improvements and meaningful functional benefits compared with natural history, with consistent and highly statistically significant results across age and disease severity. UX111 was well-tolerated and the safety profile remains favorable. “These data continue to demonstrate a remarkable and unprecedented separation from the natural history of Sanfilippo syndrome through more than eight years of follow-up, w
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- Ultragenyx Pharmaceutical Inc. Sued for Securities Law Violations - Contact the DJS Law Group to Discuss Your Rights – RAREBusiness Wire
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