학술논문

Feasibility and utility of in-home body weight support harness system use in young children treated for spinal muscular atrophy: A single-arm prospective cohort study.
Document Type
article
Source
PLoS ONE, Vol 19, Iss 3, p e0300244 (2024)
Subject
Medicine
Science
Language
English
ISSN
1932-6203
Abstract
PurposeThis single-arm prospective cohort study aimed to evaluate the feasibility and utility of in-home body weight support harness system (BWSS) use in children treated for spinal muscular atrophy (SMA).MethodsIndividuals with 2 or 3 copies of SMN2 who received pharmacotherapeutic treatment, had head control, and weight ResultsAll 32 participants (2.9 (SD 1.9) yrs), improved or remained stable on all outcomes. Average reported frequency of use was 4.1(2.3) hrs/week. Controlling for other covariates, frequency of use explained over 70% of the variability in change scores. Family feedback was overwhelmingly positive.ConclusionUse of in-home BWSS is a safe, feasible and useful option to increase exercise dosage after treatment in SMA and may help optimize motor abilities.Trial registrationStudy registered with: Clinicaltrials.gov Clinicaltrials.gov identifier: NCT05715749.