A qualitative, mixed-method approach to reaching consensus on function, fatigue, and fatigability outcomes in teens and adults living with spinal muscular atrophy

SMA key opinion leaders and adults with SMA agreed that ADLs would be sensitive and impactful outcomes in the assessment of function, perceived fatigue, and perceived fatigability. The findings of this study form a foundation for reaching consensus around a core set of outcome measures for assessing disease status, perceived fatigue, and perceived fatigability in teens and adults with SMA in the U.S.

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Publication Details

Journal
Orphanet Journal of Rare Diseases
Published
2025-10-17
DOI
https://doi.org/10.1186/s13023-025-04047-x
Primary Topic
Neurogenetic and Muscular Disorders Research
Type
article
Field-Weighted Citation Impact
0.00

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article

A qualitative, mixed-method approach to reaching consensus on function, fatigue, and fatigability outcomes in teens and adults living with spinal muscular atrophy

Mary Ann Curry, Lisa Belter, Jessica J. Tingey, Jacqueline Glascock et al.
Orphanet Journal of Rare Diseases
Neurogenetic and Muscular Disorders Research
article

A qualitative, mixed-method approach to reaching consensus on function, fatigue, and fatigability outcomes in teens and adults living with spinal muscular atrophy

Mary Ann Curry, Lisa Belter, Jessica J. Tingey, Jacqueline Glascock, Meghan Moore Burk
article en

Abstract

SMA key opinion leaders and adults with SMA agreed that ADLs would be sensitive and impactful outcomes in the assessment of function, perceived fatigue, and perceived fatigability. The findings of this study form a foundation for reaching consensus around a core set of outcome measures for assessing disease status, perceived fatigue, and perceived fatigability in teens and adults with SMA in the U.S.

Orphanet Journal of Rare DiseasesVol. 20(1)
Children's Hospital Colorado (US), Cure Spinal Muscular Atrophy (US), University of Colorado Denver (US)
Biogen, Children's Hospital of Philadelphia, Ohio State University, Children's Hospital Colorado, University of Minnesota, Nationwide Children's Hospital, Johns Hopkins University, Genentech
Openalex Percentile: Top 59%
Neurogenetic and Muscular Disorders Research
DOIOpen Access PDFOpenAlex
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A qualitative, mixed-method approach to reaching consensus on function, fatigue, and fatigability outcomes in teens and adults living with spinal muscular atrophy — Mary Ann Curry, Lisa Belter, et al. · Orphanet Journal of Rare Diseases (2025) | TGRS Research Map | TGRS