MUSCULAR DYSTROPHIES

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

This study tested a new medicine called AOC 1020 in 90 adults with FSHD, a genetic muscle-weakening disease. The goal was to check if the drug is safe and to measure how it moves through the body. Participants received either the drug or a placebo by IV, and researchers looked fo…

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 a powered soft exoskeleton (a wearable robotic suit) on 32 adults with various muscle disorders like muscular dystrophy. The goal was to see if the device is safe and can improve walking. Participants performed walking tests with and without the suit to measure …

This study looked at 30 people with a type of muscular dystrophy (FSHD1) to measure levels of inflammatory markers in their blood. Researchers compared these levels to healthy controls to see if inflammation plays a role in muscle damage. The goal was to better understand the dis…

This study involved 116 people with Limb Girdle Muscular Dystrophy (LGMD), a group of rare genetic disorders causing progressive shoulder and hip muscle weakness. Researchers measured changes in mobility, muscle function, breathing, and daily activities over time. The goal was to…

This study tested a wearable robotic leg device (Keeogo Dermoskeleton) in 50 adults with various muscle disorders like muscular dystrophy. The goal was to see if it is safe and can improve walking in a single session. Researchers measured walking tests with and without the device…