Long-Term Open-Label Study Evaluating Oral Miglustat Treatment in Patients With Neuronal Ceroid Lipofuscinosis Type 3
Miglustat showed a favorable safety profile and was associated with a slower rate of physical decline compared with historical controls. Limitations include small sample size, genetic heterogeneity, and open-label design.
Authors
- Nicola Pietrafusa (ORCID: https://orcid.org/0000-0003-2446-796X)
- Tiziana Corsetti
- Costanza Calabrese (ORCID: https://orcid.org/0000-0002-8430-4508)
- Marina Trivisano (ORCID: https://orcid.org/0000-0002-9841-8581)
- Angela De Dominicis (ORCID: https://orcid.org/0000-0002-8526-1511)
- Leonardo Vallesi
- Cinzia Correale (ORCID: https://orcid.org/0000-0002-9302-5412)
- Nicola Specchio (ORCID: https://orcid.org/0000-0002-8120-0287)
- Simona Cappelletti (ORCID: https://orcid.org/0000-0003-0738-1240)
- Licia Salimbene
Institutions
- Bambino Gesù Children's Hospital (IT)
- KU Leuven (BE)
Publication Details
- Journal
- Neurology
- Published
- 2025-09-09
- DOI
- https://doi.org/10.1212/wnl.0000000000214110
- Citations
- 2
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 2.33