Development of trofinetide for the treatment of Rett syndrome: from bench to bedside

Development of trofinetide for the treatment of Rett syndrome: from bench to bedside

Rett syndrome (RTT) is rare neurodevelopmental disorder caused by mutations in the MECP2 gene that encodes methyl-CpG-binding protein 2 (MeCP2), a DNA-binding protein with roles in epigenetic regulation of gene expression. Functional loss of MeCP2 results in abnormal neuronal maturation and plastici...

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Bibliographic Details
Main Authors: Melissa Kennedy, Larry Glass, Daniel G. Glaze, Steve Kaminsky, Alan K. Percy, Jeffrey L. Neul, Nancy E. Jones, Daniela Tropea, Joseph P. Horrigan, Paige Nues, Kathie M. Bishop, James M. Youakim
Format: Article
Language:English
Published: Frontiers Media S.A. 2024-01-01
Series:Frontiers in Pharmacology
Subjects:
trofinetide
Rett syndrome
Neuren Pharmaceuticals
Acadia Pharmaceuticals
International Rett syndrome Foundation
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Online Access:https://www.frontiersin.org/articles/10.3389/fphar.2023.1341746/full

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https://www.frontiersin.org/articles/10.3389/fphar.2023.1341746/full

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