Safety and efficacy of olesoxime in patients with type 2 or non-ambulatory type 3 spinal muscular atrophy: a randomised, double-blind, placebo-controlled phase 2 trial -

Safety and efficacy of olesoxime in patients with type 2 or non-ambulatory type 3 spinal muscular atrophy: a randomised, double-blind, placebo-controlled phase 2 trial
Information type: Randomised controlled trials
Source: The Lancet Neurology
Specialities: Neurological disorders
Summary
This study (n=160) did not report a statistically significant effect with olexisome vs placebo in the primary outcome (change from baseline to 24 months in functional domains 1 and 2 of the Motor Function Measure): 0.18 vs −1.82 respectively (treatment difference = 2, p=0.0676).
UKMi comment
Olesoxime prevents excessive permeability of the mitochondrial membrane under stress conditions, preventing apoptosis by reducing the release of pro-apoptotic factors and maintaining energy production.
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