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 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 caregiver concerns to guide the development and selection of appropriate clinical trial outcome measures for these disorders.

Methods

Caregivers of participants enrolled in the US Natural History Study of RTT and RTT-related disorders (n = 925) were asked to identify the top 3 concerning problems impacting the affected participant. We

SUBMITTER: Neul JL 

PROVIDER: S-EPMC10571464 | biostudies-literature | 2023 Oct

REPOSITORIES: biostudies-literature