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 Hospital, Basel, Switzerland, children with RS were included if they were older than 6 years and met the established diagnostic criteria of RS, including a positive MeCP2 mutation. Participants were observed 6 months before and after treatment and received 12 months of fingolimod treatment. Serum samples of 50 children without RS served as reference for brain-derived neurotrophic factor (BDNF) measurements. Primary outcome measures were safety and efficacy, the latter measured by change in levels

SUBMITTER: Naegelin Y 

PROVIDER: S-EPMC7789265 | biostudies-literature | 2021 Jan

REPOSITORIES: biostudies-literature