International Niemann–Pick Disease Alliance
Vtesse, Inc. have announced the preliminary results of the open-label Phase 1 clinical trial of VTS-270 (a formulation of (2-hydroxypropyl)-beta-cyclodextrin) for the treatment of Niemann-Pick Disease Type C (NPC) conducted by researchers at the National Institutes of Health (NIH) Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), in Maryland, USA.
Preliminary analyses, conducted post-hoc, suggest that the rate of disease progression had slowed down (based on a standardised measure) in children treated with VTS-270 in the Phase 1 trial as compared to the rate in an age- and disease severity-matched cohort obtained from a separate natural history study of NPC patients. The analyses also show that children treated with VTS-270 demonstrated improvement on several disease domains.
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