RecruitingNCT06955897

Characterizing Perceived Physical Fatigability in Nusinersen-treated SMA

Characterizing Perceived Physical Fatigability in Nusinersen-treated Adolescents and Adults: The SMA EFFORT

Sponsor

Columbia University

Enrollment

45 participants

Start Date

Apr 24, 2025

Study Type

OBSERVATIONAL

Conditions

Spinal Muscular Atrophy

Summary

The purpose of this project is to investigate the utility of the SMA EFFORT, an SMA-specific patient-reported outcome measure, to assess perceived physical fatigability that is anchored to intensity and duration of activities. We aim to characterize perceived physical fatigability (PPF) in a diverse cohort of people with SMA (pwSMA) and evaluate the change of PPF before and after nusinersen dosing.

Eligibility

Min Age: 12 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying Observational for people with spinal muscular atrophy. The study is currently recruiting participants at 1 location. People eligible for this study include aged 12 Years and older.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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