Pilot trial on the efficacy and safety of pantethine in children with pantothenate kinase-associated neurodegeneration: a single-arm, open-label study

OBJECTIVE: This study aimed to explore the efficacy and safety of pantethine in children with pantothenate kinase-associated neurodegeneration (PKAN). METHODS: A single-arm, open-label study was conducted. All subjects received pantethine during the 24-week period of treatment. The primary endpoints...

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Detalles Bibliográficos
Autores principales: Chang, Xuting, Zhang, Jie, Jiang, Yuwu, Yao, Bufan, Wang, Jingmin, Wu, Ye
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2020
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7491170/
https://www.ncbi.nlm.nih.gov/pubmed/32928263
http://dx.doi.org/10.1186/s13023-020-01530-5

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