NEUROMUSCULAR DISEASES

This study tested a drug called ataluren in 360 boys (age 5 and older) with Duchenne muscular dystrophy caused by a specific genetic mistake called a nonsense mutation. The goal was to see if ataluren could help them walk farther over 72 weeks compared to a placebo. All participa…

This study tested a drug called apitegromab in 188 people aged 2 to 21 with later-onset spinal muscular atrophy (SMA types 2 and 3) who were already taking nusinersen or risdiplam. The goal was to see if adding apitegromab could improve muscle function more than a placebo. Partic…

This study looked at the long-term safety of a drug called AOC 1001 in adults with myotonic dystrophy type 1 (DM1), a genetic muscle disease. 37 people who completed an earlier study received multiple doses of the drug by IV. Researchers tracked side effects and measured drug lev…

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 who also care for someone with the same condition. Th…

This study tested whether the EXOPULSE Mollii suit, which delivers mild electrical pulses, can reduce pain, fatigue, and improve quality of life in people with fibromyalgia. Twenty-eight adults wore either the active suit or a sham (inactive) suit for two weeks. The goal was to s…

This study looked at how muscles use oxygen during exercise in people with different neuromuscular diseases compared to healthy volunteers. Seventeen participants did a knee exercise while a device measured muscle oxygen levels. The goal was to understand if oxygen problems are l…

This study checked muscle activity in 42 male football players, half with a recent hamstring strain. Using special sensors during exercises, researchers found that injured players still had altered muscle use in their hamstrings, buttocks, back, and lower spine. These lasting cha…