Subgroup and Subdomain Analysis of Patients with Long Term Follow-Up In a Phase III, Randomized, Placebo-Controlled Crossover Trial with Levacetylleucine (IB1001) for Niemann-Pick Disease Type C (S31.004)
To evaluate the efficacy of levacetylleucine compared to placebo across subdomains of the modified Disability Rating Scale (mDRS), in adult and pediatric subgroups within the Scale for the Assessment and Rating of Ataxia (SARA) and investigate long-term findings from IB1001-301.
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)
- Jean-François de Raymond
- Bethany Zanrucha
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.0000000000208822
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00