Randomized Trialhigh evidence
Safety and efficacy of levacetylleucine in ataxia-telangiectasia: a phase 3, randomised, double-blind, placebo-controlled crossover trial.
The Lancet. Neurology·July 1, 2026·PMID 42309084
Why it mattersAtaxia-telangiectasia is a rare neurodegenerative disorder with no approved disease-modifying therapy. This trial provides high-level evidence for a symptomatic treatment that improves motor function, addressing a critical unmet need.
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