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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
'It's impressive to see how our study patients treated with UX111 have maintained their communication skills despite being the age in which regression begins to occur,' stated
Following treatment with UX111 (3x1013 vg/kg), levels of CSF-HS decreased within the first month post treatment in all patients. 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
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