This observational study aims to understand how people with Spinal Muscular Atrophy (SMA) who are treated with nusinersen experience physical fatigability (feeling tired or weak after activity). Researchers will use a special questionnaire called SMA EFFORT to measure how you feel when doing daily physical activities. They want to see how this feeling changes before and after nusinersen doses. You may be able to join if you are 12 years or older, have any type of 5qSMA, and have been receiving a stable dose of nusinersen for at least six months. The study is currently unclear on its recruitment status and plans to enroll 45 participants.

Study design

This is an observational study with an unclear status, planning to enroll 45 participants. It is not specified if it is randomized, blinded, or what phase it is in.

What's involved

You would complete the SMA EFFORT questionnaire at baseline, 1 week, and 1 month. The study does not specify other visits or procedures.

Compensation

Not stated in the trial record.

Follow-up

Participants will be followed for at least one month, based on the primary endpoint measurements.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.