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Fingolimod in children with Rett syndrome: the FINGORETT study
BACKGROUND: Rett syndrome (RS) is a severe neurodevelopmental disorder for which there is no approved therapy. This study aimed to assess safety and efficacy of oral fingolimod in children with RS using a pre-post and case–control design. METHODS: At the University of Basel Children’s Hospital, Base...
Autores principales: | Naegelin, Yvonne, Kuhle, Jens, Schädelin, Sabine, Datta, Alexandre N., Magon, Stefano, Amann, Michael, Barro, Christian, Ramelli, Gian Paolo, Heesom, Kate, Barde, Yves-Alain, Weber, Peter, Kappos, Ludwig |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BioMed Central
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7789265/ https://www.ncbi.nlm.nih.gov/pubmed/33407685 http://dx.doi.org/10.1186/s13023-020-01655-7 |
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