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June 9, 2026Neurology

Long-Term 30-Month Results of N-Acetyl-L-Leucine for Niemann-Pick Disease Type C (P11-7.003)

Michael Strupp Marc Patterson Bethany Zanrucha Janelle Raymond Asante Hatcher Jorgji Kerthi Taylor Fields Ian Billington Tatiana Brémovà-Ertl

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Key Points

The aim is to evaluate how N-acetyl-L-leucine affects individuals with Niemann-Pick disease type C over a long duration.
Evaluated participants with Niemann-Pick disease type C over a 30-month period.
Administered N-acetyl-L-leucine as an intervention.
N-acetyl-L-leucine showed positive long-term effects in Niemann-Pick disease type C.
Significant improvements in specific neurological functions were observed.

Implication

Evaluates long-term effects of N-acetyl-L-leucine in Niemann-Pick disease type C, indicating potential benefits.

Abstract

To evaluate the long-term effects of N-acetyl-L-leucine (NALL) in people with Niemann-Pick disease type C (NPC).

Discussion

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Cite This Study

Strupp et al. (2026) studied this question.

synapsesocial.com/papers/6a2cf34dfaef96ed7f055d79 https://doi.org/10.1212/wnl.0000000000216341

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Keep reading

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
2Results of a Phase III, Randomized, Placebo-controlled Crossover Trial with N-acetyl-L-leucine for Niemann-Pick Disease Type C (PL5.001)2024 · 1 citations
3Stereospecific rapid activation of Transcription Factor EB (TFEB) by Levacetylleucine (NALL)Original Research · 2025 · 5 citations
4N-acetyl-L-leucine in Neurological Disorders: Mechanisms, Evidence, and Therapeutic Perspectives.Human Clinical Trial · 2026
5Therapeutic Interventions in Niemann–Pick Disease Type C: A Systematic Review of Clinical Outcomes and SafetySystematic Review · 2026

Results from a Global Phase 3 Trial (IB1001-303) Evaluating N-acetyl-L-leucine in Ataxiatelangiectasia (P11-7.004)2026
N-acetyl-L-leucine normalizes Transcription Factor EB activity by stereospecific bidirectional modulation in a HeLa cell model of Niemann-Pick disease type C2026
Long-term efficacy of intrathecal cyclodextrin in patients with Niemann-Pick disease type C2024
Blood-Based Biomarkers as Predictors of All-Cause DementiaNeurology · 2026
Antidopaminergic Medications and Clinical Outcomes in Huntington DiseaseNeurology · 2026

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