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...

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Dettagli Bibliografici
Pubblicato in:Orphanet J Rare Dis
Autori principali: 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
Natura: Artigo
Lingua:Inglês
Pubblicazione: BioMed Central 2021
Soggetti:
Research
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC7789265/
https://ncbi.nlm.nih.gov/pubmed/33407685
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-020-01655-7
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