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) has enabled development of novel therapeutic approaches that are currently undergoing clinical evaluation or are proposed to move into clinical development. Clinical trial success depends on outcome measures that assess clinical features that are most impactful for affected individuals. To determine the top concerns in RTT and RTT-related disorders we asked caregivers to list the top clinical concerns in order to gain information to guide the development and selection of outcome measures for future clinical trials.

Methods

Caregivers of participants enrolled in the US Natural History Study of RTT and related disorders were asked to identify the top 3 concerning problems

SUBMITTER: Neul JL 

PROVIDER: S-EPMC10055548 | biostudies-literature | 2023 Mar

REPOSITORIES: biostudies-literature