Randomized trial evaluates efficacy and safety of N-acetyl-L-leucine in Ataxia-Telangiectasia, suggesting therapeutic potential.
To assess the efficacy and safety of N-acetyl-L-leucine (NALL) in people with Ataxia-Telangiectasia (A-T) in a global, phase 3, randomized, placebo-controlled, double-blind crossover trial (IB1001-303).
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Patterson et al. (2026) studied this question.
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