Top caregiver concerns in Rett syndrome and related disorders: data from the US natural history study

Top caregiver concerns in Rett syndrome and related disorders: data from the US natural history study

Abstract Objective Recent advances in the understanding of neurodevelopmental disorders such as Rett syndrome (RTT) have enabled the discovery of novel therapeutic approaches that require formal clinical evaluation of efficacy. Clinical trial success depends on outcome measures that assess clinical...

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Bibliographic Details
Main Authors: Jeffrey L. Neul, Timothy A. Benke, Eric D. Marsh, Bernhard Suter, Lori Silveira, Cary Fu, Sarika U. Peters, Alan K. Percy, for the Rett syndrome Natural History Study Group
Format: Article
Language:English
Published: BMC 2023-10-01
Series:Journal of Neurodevelopmental Disorders
Subjects:
Rett syndrome
CDKL5
FOXG1
MECP2 duplication
Neurodevelopmental disorders
Caregiver concerns
Online Access:https://doi.org/10.1186/s11689-023-09502-z

Internet

https://doi.org/10.1186/s11689-023-09502-z

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