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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...
Autores principales: | , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BioMed Central
2020
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Materias: | |
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 |