Family and caregiver perspectives on gene therapy for Rett syndrome

Family and caregiver perspectives on gene therapy for Rett syndrome

Introduction: Rett syndrome (RTT) is a neurodevelopmental disorder that primarily affects females and can result in cognitive impairment, seizures, spasticity, breathing problems, gastrointestinal issues, motor impairment, and behavioral concerns. Gene therapy may be a potential treatment in the fut...

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Bibliographic Details
Main Authors: Keri Ramsey, Madison LaFleur, Kiana Robinson, Mark Borgstrom, Ashley Ryan, Vinodh Narayanan, Valerie Schaibley
Format: Article
Language:English
Published: Elsevier 2024-01-01
Series:Rare
Subjects:
Rett syndrome
Gene therapy
Risk
Caregiver
Online Access:http://www.sciencedirect.com/science/article/pii/S2950008724000280
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