Pilot trial on the efficacy and safety of pantethine in children with pantothenate kinase-associated neurodegeneration: a single-arm, open-label study | Orphanet Journal of Rare Diseases | Full Text

Pilot trial on the efficacy and safety of pantethine in children with pantothenate kinase-associated neurodegeneration: a single-arm, open-label study | Orphanet Journal of Rare Diseases | Full Text

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

This study aimed to explore the efficacy and safety of pantethine in children with pantothenate kinase-associated neurodegeneration (PKAN).

Authors:Xuting Chang, Jie Zhang, Yuwu Jiang, Bufan Yao, Jingmin Wang and Ye Wu

Citation:Orphanet Journal of Rare Diseases 2020 15:248

Content type:Research

 

Published on: 14 September 2020

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