MYOTONIC DYSTROPHY

This study tests a new medicine, DYNE-101, for adults with myotonic dystrophy type 1, a condition that causes muscle weakness and stiffness. About 150 people will receive either the drug or a placebo for 48 weeks. The goal is to see if the drug improves muscle function, like stan…

This study tests a new gene therapy called SAR446268 for people aged 10 to 55 with myotonic dystrophy type 1, a genetic muscle disease. The therapy aims to reduce harmful RNA levels and improve muscle function. The trial has two parts: first, finding the safest dose, then testing…

This study is testing a blood test that can check for genetic disorders in unborn babies without the risks of traditional invasive tests. The test looks at fetal DNA found in the mother's blood as early as 9 weeks into pregnancy. Researchers aim to make this safe, accurate diagno…

This study tests whether a once-daily pill called mexiletine can safely reduce muscle stiffness (myotonia) in people with myotonic dystrophy types 1 and 2. About 176 adults aged 16 and older will take the drug or a placebo for 26 weeks. The main goal is to see if handgrip relaxat…

This study is not testing a treatment. Instead, it aims to develop a better way to measure muscle changes in people with myotonic dystrophy using MRI scans. Researchers will enroll 75 adults (ages 18-65) with or without the condition to see if muscle imaging can serve as a reliab…

This study is looking for easier ways to track muscle diseases like muscular dystrophy. Instead of taking small pieces of muscle (biopsies), researchers want to see if blood and urine samples, plus painless ultrasound and electrical tests on arms and legs, can give the same infor…

This study creates a national registry for people with myotonic dystrophy (DM) or facioscapulohumeral muscular dystrophy (FSHD) and their family members. The goal is to connect patients with researchers to speed up understanding of these inherited muscle-weakening diseases. Parti…

This study looks for markers in blood and urine that can show how active and severe myotonic dystrophy is. The goal is to find a less invasive way to measure the disease than taking a piece of muscle (biopsy). Researchers will compare samples from 215 people with and without the …

This study tests a portable digital device that analyzes walking without markers, comparing it to standard motion-capture systems. It includes 30 adults (ages 18-65) with certain neuromuscular diseases or healthy volunteers. The goal is to see if this simpler tool can reliably me…