Trofinetide for the treatment of Rett syndrome: Long-term safety and efficacy results of the 32-month, open-label LILAC-2 study-Reference-Cited by-同舟云学术

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Trofinetide for the treatment of Rett syndrome: Long-term safety and efficacy results of the 32-month, open-label LILAC-2 study

Published:2024-07 Issue: Volume: Page:
ISSN:2666-6340
Container-title:Med
language:en
Short-container-title:Med

Author:

Percy Alan K.,Neul Jeffrey L.,Benke Timothy A.,Berry-Kravis Elizabeth M.,Glaze Daniel G.,Marsh Eric D.,Barrett Amy M.,An Di,Bishop Kathie M.,Youakim James M.

Funder

ACADIA Pharmaceuticals Inc

Publisher

Elsevier BV

Reference23 articles.

1. Global prevalence of Rett syndrome: systematic review and meta-analysis;Petriti;Syst. Rev.,2023

2. Rett syndrome is caused by mutations in X-linked MECP2, encoding methyl-CpG-binding protein 2;Amir;Nat. Genet.,1999

3. Developmental and maintenance defects in Rett syndrome neurons identified by a new mouse staging system in vitro;Baj;Front. Cell. Neurosci.,2014

4. Defects during Mecp2 null embryonic cortex development precede the onset of overt neurological symptoms;Bedogni;Cereb. Cortex,2016

5. Widespread changes in dendritic and axonal morphology in Mecp2-mutant mouse models of Rett syndrome: evidence for disruption of neuronal networks;Belichenko;J. Comp. Neurol.,2009

Cited by 1 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Noncoding variants; trofinetide; microglia shapes;The Transmitter;2024

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