DUCHENNE MUSCULAR DYSTROPHY

This program offers continued treatment with vamorolone, a potential steroid alternative, to boys with Duchenne muscular dystrophy who finished earlier studies. It aims to keep them on the drug while the company prepares its FDA application. Participants must have completed speci…

This study tests a single intravenous dose of SGT-003, a gene therapy designed to help boys with Duchenne muscular dystrophy produce a shortened version of the dystrophin protein. About 60 males from infancy to under 18 years old will take part. Researchers will monitor safety an…

This study looks at two add-on treatments for Duchenne muscular dystrophy (DMD): a drug called tadalafil that improves blood flow, and aerobic exercise training. The goal is to see if these can improve muscle function and delay disease progression. About 50 ambulatory children wi…

This study tests an oral drug called SAT-3247 in 51 boys aged 7 to 10 with Duchenne muscular dystrophy (DMD). The goal is to see if it can improve muscle strength and function over 12 weeks. Participants will take the drug or a placebo on weekdays, and all will continue their usu…

This early-stage trial tests whether injecting specially grown muscle cells into the feet of adults with Duchenne muscular dystrophy is safe. Only 8 non-ambulatory participants will receive the cells, made from stem cells in a lab. The main goal is to find a safe dose, not yet to…

This study looks at the long-term safety and effectiveness of the drug givinostat in people with Duchenne muscular dystrophy (DMD) who have already taken part in earlier givinostat studies. About 206 participants will take the drug for an extended period to monitor side effects a…

This trial tests a one-time gene therapy called RGX-202 for boys with Duchenne muscular dystrophy (DMD), a severe muscle-weakening disease. The therapy delivers a gene to help muscles produce a shortened but functional version of a key protein. The study involves about 65 boys ag…

This study tests a single intravenous dose of SGT-003, a gene therapy, in 80 boys with Duchenne muscular dystrophy who can still walk. The goal is to see if it improves muscle function, like how fast they can stand up or climb stairs. Participants will receive either the therapy …

This study follows 250 boys aged 2 and older with Duchenne muscular dystrophy who are taking vamorolone (AGAMREE). Researchers will monitor side effects, growth, bone health, heart function, and quality of life over time. The goal is to better understand the long-term safety and …

This early-stage study tests a single injection of a gene therapy called INS1201 in 12 young boys (ages 2 to 5) with Duchenne muscular dystrophy who can still walk. The main goal is to see if the treatment is safe and where it goes in the body. Researchers will also look for sign…

This study tests a drug called WVE-N531 in people with Duchenne muscular dystrophy (DMD), a genetic condition that causes muscle weakness. The drug aims to help the body produce a protein called dystrophin, which is missing in DMD. About 26 participants will receive the drug thro…

This study tests a weekly intravenous drug, NS-089/NCNP-02, in 20 boys aged 4 to 14 with Duchenne muscular dystrophy (DMD) who have a specific genetic mutation. The goal is to help their bodies produce a missing muscle protein, dystrophin, to slow muscle damage. Participants must…

This study tests a new medicine, BMN 351, in 18 boys aged 4 to 10 with Duchenne muscular dystrophy who have a specific genetic change. The main goal is to check if the drug is safe and how the body handles it. Participants receive multiple doses through an IV, and researchers wil…

This study tests whether a fun, game-based occupational therapy program can improve daily living skills, satisfaction, and emotional well-being in 20 adolescents with Duchenne muscular dystrophy. Participants will use a computer or tablet at home, and researchers will compare res…

This study screens males with Duchenne muscular dystrophy (ages 0 to under 25) for antibodies against AAV8, a virus used in some gene therapies. The goal is to find out how many have these antibodies and identify those who might be eligible for future gene therapy trials. No trea…

This study uses wearable monitors (glucose sensors, heart rate patches, and activity trackers) to see if high blood sugar is linked to heart problems in people with Duchenne muscular dystrophy. Researchers will compare 80 participants with and without Duchenne. The goal is to fin…

This study uses MRI scans to monitor muscle damage and fat buildup in boys and men with Duchenne or Becker muscular dystrophy over 5-10 years. Researchers compare these images with healthy individuals and link them to daily activities like walking. The goal is to develop better w…

This study follows 220 boys aged 4 to 9 with Duchenne muscular dystrophy to learn how the disease naturally progresses over 6 to 36 months. Researchers will measure muscle strength, walking ability, heart and lung function, and quality of life. The goal is to collect data that ca…

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 an online registry for people with Duchenne or Becker muscular dystrophy, including female carriers. Participants share their health information to help researchers learn more about the diseases and to connect with clinical trials. The goal is to improve care a…

This study looks for markers in blood and urine that can show how active and severe Duchenne muscular dystrophy is. The goal is to find a less invasive way to track the disease instead of taking muscle biopsies. Researchers will compare samples from people with Duchenne or Becker…

This study will use wearable monitors to track blood sugar levels and heart rate in 10 people with Duchenne muscular dystrophy (DMD) over two years. The goal is to see if high blood sugar and changes in heart rate are linked to heart problems in DMD. Participants will also have h…