DUCHENNE MUSCULAR DYSTROPHY

This study tests a one-time gene therapy called RGX-202 for boys with Duchenne muscular dystrophy (DMD). The therapy aims to help muscles work better by delivering a special protein. About 65 boys aged 1 to 12 will take part. The main goals are to check safety and see if the ther…

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 their ability to stand up, walk, and climb stairs compared to a placebo. All participants will receive the ther…

This study looks at the long-term safety and effects of the drug Givinostat in people with Duchenne muscular dystrophy (DMD) who have already taken it in earlier studies. About 206 participants will take the drug and be monitored for side effects and how well the drug works over …

This study tests an oral drug called SAT-3247 in about 51 boys aged 7 to 10 with Duchenne muscular dystrophy (DMD). The goal is to find the best dose and see if it is safe and helps muscle strength. Participants will take the drug or a placebo for 12 weeks, and all will continue …

This study monitors the long-term safety of the drug vamorolone (AGAMREE) in boys aged 2 and older with Duchenne muscular dystrophy. Researchers will track growth, bone health, heart function, and quality of life over time. The goal is to better understand the drug's risks and be…

This program offers boys with Duchenne muscular dystrophy (DMD) in the US and Canada continued access to the experimental drug vamorolone after they finish other related studies. The goal is to keep providing treatment while the drug is being reviewed for approval. Participants m…

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 early-stage trial tests whether injecting lab-grown muscle cells into the feet of adults with Duchenne muscular dystrophy is safe. Only 8 non-ambulatory participants will receive the cells, and researchers will monitor for side effects and immune reactions. The goal is to fi…

This study tests a single intravenous dose of SGT-003, a gene therapy designed to help boys with Duchenne muscular dystrophy produce a shortened but functional version of the dystrophin protein. About 60 boys aged 0 to 18 will receive the treatment and be followed for 5 years. Th…

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, compared to standard therapy. Participants must be able to use a computer or tablet …

This study follows 220 boys aged 4 to 9 with Duchenne muscular dystrophy for up to 3 years. Researchers will collect data on muscle strength, walking, breathing, and heart function to better understand how the disease naturally progresses. No new treatments are tested; the goal i…

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 aims to find less invasive ways to track muscular dystrophy, replacing painful muscle biopsies with simple blood and urine tests plus painless ultrasound and electrical muscle scans. Researchers will compare these new methods in 465 people with different types of muscu…

This study uses MRI scans to measure 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 volunteers and track how changes relate to daily activities like walking. The goal is to de…

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 is a simple blood test to check if boys and young men with Duchenne muscular dystrophy have antibodies against a virus used in gene therapy. It aims to find who might be eligible for future gene therapy trials. Up to 200 participants aged 0 to under 25 will be enrolled…

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…