SARA Eight-Item Subgroup Analysis of N-acetyl-L-leucine for Niemann-Pick Disease Type C (S27.002)
To evaluate the efficacy of N-acetyl-L-leucine (NALL) compared to placebo within each of the eight items of the Scale for the Assessment and Rating of Ataxia (SARA).
Authors
- Marc C. Patterson (ORCID: https://orcid.org/0000-0002-1116-126X)
- Tatiana Brémovà-Ertl (ORCID: https://orcid.org/0000-0002-7397-045X)
- Ian Billington
- Michael Leo Strupp (ORCID: https://orcid.org/0000-0002-9544-2279)
- Bethany Zanrucha
- Janelle Schafer
Institutions
- University Hospital of Bern (CH)
- Ludwig-Maximilians-Universität München (DE)
Publication Details
- Journal
- Neurology
- Published
- 2025-04-07
- DOI
- https://doi.org/10.1212/wnl.0000000000209037
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00