NEUROMUSCULAR DISEASES

This study tested a drug called ataluren in 360 boys with Duchenne muscular dystrophy caused by a specific genetic mistake (nonsense mutation). The main goal was to see if ataluren could help them walk farther over 72 weeks compared to a placebo. All participants were also taking…

This Phase 3 trial tested apitegromab, a drug that blocks a muscle-limiting protein, in 188 nonambulatory children and young adults with later-onset spinal muscular atrophy (SMA types 2 and 3). All participants were already taking standard SMA therapies (nusinersen or risdiplam).…

This study is a follow-up to an earlier trial, testing the long-term safety and effects of a drug called AOC 1001 in adults with myotonic dystrophy type 1 (DM1), a genetic muscle disease. 37 participants who completed the first study received multiple doses of AOC 1001 by IV infu…

This study tested whether virtual peer support helps family caregivers of people with neuromuscular disease feel more in control and less isolated. About 100 Canadian caregivers joined online sessions with trained peer mentors. The goal was to improve caregivers' sense of mastery…

This study tested whether a special suit called EXOPULSE Mollii can help adults with fibromyalgia feel less pain, fatigue, and depression. Twenty-eight participants wore either the active suit or a sham (fake) suit for two weeks, then had a washout period. After that, everyone co…

This study tests whether digital tools like smartphone apps, wearable sensors, and home spirometers can reliably track symptoms of neuromuscular diseases. Researchers will compare data collected at home with in-clinic measurements from 40 participants aged 12–60. The goal is to d…

This study tested a new sensor placed inside the nose to measure carbon dioxide levels in 21 people with breathing conditions like COPD or obesity-related breathing problems. The goal was to see if the sensor is safe, easy to use, and gives accurate readings compared to standard …

This completed study looked at how muscle oxygen levels change during exercise in people with various neuromuscular diseases compared to healthy volunteers. Seventeen participants did a knee-extension exercise while a device measured muscle oxygen. The goal was to understand diff…

This study looked at 42 male football players, half with a recent hamstring strain and half without. Using muscle activity sensors, researchers found that injured players still had weaker activation in several muscles—not just the hamstring—up to six months later. These hidden de…