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April 9, 2024Neurology

Results of a Phase III, Randomized, Placebo-controlled Crossover Trial with N-acetyl-L-leucine for Niemann-Pick Disease Type C (PL5.001)

Tatiana Brémovà-Ertl Taylor Fields Mallory Factor Κyriakos Martakis Michael Strupp

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Abstract

We conducted a Phase 3 trial to confirm the safety and efficacy of the active L-enantiomer, N-acetyl-L-leucine (NALL) for pediatric (≥ 4 years) and adult patients with NPC.

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Cite This Study

Brémovà-Ertl et al. (2024) studied this question.

synapsesocial.com/papers/68e6fdb8b6db6435876781c3 https://doi.org/10.1212/wnl-.0000000000205236

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Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

1Disease-Modifying, Neuroprotective Effect of N-Acetyl- l -Leucine in Adult and Pediatric Patients With Niemann-Pick Disease Type CRCT · 2025 · 14 citations
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