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)
These data are from the modified intention to treat group (mITT) in the pivotal Transpher A study (N=17).
- These data are from the modified intention to treat group (mITT) in the pivotal Transpher A study (N=17).
- 8 of 17 patients in the mITT group who reached 24 months post-treatment achieved an overall mean percent reduction from baseline of 51% (p
- Specifically, 15 of 17 patients had both a reduction in toxic CSF HS exposure and an improvement in cognitive function.
- Both trials are ongoing, and patients will continue to be followed for a minimum of 5 years following treatment with UX111.