Facioscapulohumeral muscular dystrophy 1

This study tests an experimental drug called ARO-DUX4 in people with facioscapulohumeral muscular dystrophy type 1 (FSHD1), a genetic condition that causes muscle weakness. The main goal is to check the drug's safety and how the body processes it. About 60 adults and teens will r…

This phase 3 trial tests an experimental drug called AOC 1020 in 200 adults with facioscapulohumeral muscular dystrophy (FSHD), a genetic condition that causes progressive muscle weakness. Participants receive either the drug or a placebo by IV infusion. The study measures muscle…

This early-stage trial tests a brain-computer interface called BrainGate for people with paralysis from conditions like ALS or spinal cord injury. A small sensor is placed in the brain to interpret movement-related signals, allowing users to control a tablet computer just by thin…

This study is testing a handheld device called mScan that uses a tiny, painless electrical current to measure muscle health. Researchers want to see if it can give similar results to an MRI, but faster and more conveniently. The study involves 150 adults with and without muscle d…

This study tests whether cycling while using a virtual park app can slow the loss of motor function in children and teens with neuromuscular diseases like muscular dystrophy. Twenty-two participants will first receive standard care for 3 months, then use the virtual park bike 3 t…

This early-stage study tests whether stem cells from umbilical cord lining can safely help people with FSHD, a genetic muscle-weakening disease. Sixteen adults will receive two doses of the cells and two doses of a placebo (saline) through an IV, in random order. The main goal is…

This study aims to learn more about rare inherited disorders that affect the brain, spinal cord, muscles, and nerves. Researchers will collect medical history, perform exams, and run genetic tests on up to 3,500 participants. No new treatments are tested; the goal is to better un…

This study creates a registry for people with facioscapulohumeral muscular dystrophy (FSHD) to share their health experiences and data. Up to 5,000 participants in the U.S. will provide information through surveys to help researchers better understand the disease and develop impr…

This study aims to find better ways to measure fatigue and walking problems in people with neuromuscular diseases like muscular dystrophy and spinal muscular atrophy. Researchers will use a wearable sensor to track physical activity for one week in daily life and during a walking…

This study follows 34 adults with FSHD over one year to see how their breathing muscles change. Researchers will use ultrasound to measure diaphragm thickness and movement, along with standard breathing tests. The goal is to find out if ultrasound can detect respiratory problems …