Fingolimod in children with Rett syndrome: the FINGORETT study

Fingolimod in children with Rett syndrome: the FINGORETT study

Abstract 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 Hospita...

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Bibliographic Details
Main Authors: Yvonne Naegelin, Jens Kuhle, Sabine Schädelin, Alexandre N. Datta, Stefano Magon, Michael Amann, Christian Barro, Gian Paolo Ramelli, Kate Heesom, Yves-Alain Barde, Peter Weber, Ludwig Kappos
Format: Article
Language:English
Published: BMC 2021-01-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Rett syndrome
Fingolimod
BDNF
Online Access:https://doi.org/10.1186/s13023-020-01655-7

Internet

https://doi.org/10.1186/s13023-020-01655-7

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