Neuren’s data in Pitt Hopkins syndrome shows promise
Australia’s Neuren Pharmaceuticals (ASX: NEU) has announced top-line results from its Phase II trial of NNZ-2591 in children with Pitt Hopkins syndrome (PTHS).
Statistically-significant improvement from baseline was observed by both clinicians and caregivers from treatment, across all four efficacy measures that were specifically designed to assess the core characteristics of PTHS.
"This underserved community has such urgent unmet need"There are no approved treatments for PTHS, despite its severely debilitating impact on the lives of patients, as well as their parents and siblings.
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