Ultragenyx Announces Data Demonstrating Treatment with UX111 Results in Significant Reduction in Heparan Sulfate Exposure in Cerebrospinal Fluid Correlated with Improved Long-term Cognitive Function in Patients with Sanfilippo Syndrome Type A (MPS IIIA)
NOVATO, Calif., Feb. 06, 2024 (GLOBE NEWSWIRE) — Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) today announced data demonstrating treatment with UX111 (ABO-102) AAV gene therapy resulted in rapid and sustained decreased levels of heparan sulfate (HS) in cerebrospinal fluid (CSF) in patients with Sanfilippo syndrome type A (MPS IIIA), and that sustained reduction in CSF HS exposure over time was correlated with improved long-term cognitive development. These data are from the modified intention to treat group (mITT) in the pivotal Transpher A study (N=17). The results of this study along with the additional data from the long-term follow-up study for these subjects will be presented at the WORLDSymposium™ 2024 20th annual research meeting taking place February 4-9 in San Diego.
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