Digital endpoints for self-administered home-based functional assessment in pediatric Friedreich's ataxia.

ABSTRACT:

Background

Friedreich's ataxia is an inherited, progressive, neurodegenerative disease that typically begins in childhood. Disease severity is commonly assessed with rating scales, such as the modified Friedreich's Ataxia Rating Scale, which are usually administered in the clinic by a neurology specialist.

Objective

This study evaluated the utility of home-based, self-administered digital endpoints in children with Friedreich's ataxia and unaffected controls and their relationship to standard clinical rating scales.

Methods

In a cross-sectional study with 25 participants (13 with Friedreich's ataxia and 12 unaffected controls, aged 6-15 years), home-based digital endpoints that reflect activities of daily living were recorded over 1 week. Domains analyzed were hand mo

SUBMITTER: Mueller A 

PROVIDER: S-EPMC8419399 | biostudies-literature | 2021 Sep

REPOSITORIES: biostudies-literature