Trofinetide for the treatment of Rett syndrome: Results from the open-label extension LILAC study-Reference-Cited by-同舟云学术

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Trofinetide for the treatment of Rett syndrome: Results from the open-label extension LILAC study

Published:2024-09 Issue:9 Volume:5 Page:1178-1189.e3
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.,An Di,Bishop Kathie M.,Youakim James M.

Funder

ACADIA Pharmaceuticals Inc

Publisher

Elsevier BV

Reference30 articles.

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

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

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

4. 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

5. Gastrointestinal and nutritional problems occur frequently throughout life in girls and women with Rett syndrome;Motil;J. Pediatr. Gastroenterol. Nutr.,2012

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1. Using Precision Medicine to Disentangle Genotype–Phenotype Relationships in Twins with Rett Syndrome: A Case Report;Current Issues in Molecular Biology;2024-08-02

2. Is trofinetide a future treatment for Rett syndrome? A comprehensive systematic review and meta-analysis of randomized controlled trials;BMC Medicine;2024-07-18

3. Improvement in symptoms of Rett syndrome with trofinetide beyond clinical trial efficacy assessments: case reports;Future Rare Diseases;2024-07-15

4. Autism interventions tailored for Black Americans; gene therapy for Rett syndrome; KATNAL2 gene;The Transmitter;2024

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