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 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 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 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 …