Duchenne muscular dystrophy

This study tests whether adding sirolimus (a drug that calms the immune system) to ELEVIDYS gene therapy can prevent serious liver injury in boys with Duchenne muscular dystrophy. About 20 participants will receive the treatment and be monitored for side effects. The goal is to m…

This phase 2 trial tests an experimental drug called KER-065 in adult and pediatric males with Duchenne Muscular Dystrophy (DMD), a genetic condition that causes progressive muscle weakness. The study includes both those who can walk and those who cannot, and all participants mus…

This study tests a one-time gene therapy called SGT-003 in 80 boys with Duchenne muscular dystrophy who can still walk. The goal is to see if it helps them move better, like standing up faster and climbing stairs. Participants will receive either the therapy or a placebo first, t…

This study follows 250 boys (ages 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 an…

This study tests a one-time gene therapy called delandistrogene moxeparvovec in 83 people with Duchenne muscular dystrophy. The goal is to see if it is safe and helps the body make dystrophin, a protein missing in Duchenne. The trial is now enrolling non-ambulatory participants (…

This trial is testing whether a lower weekly dose of the corticosteroid prednisolone (5 mg/kg per week) can be as effective as the standard higher dose (10 mg/kg per week) in preserving motor function in young boys with Duchenne muscular dystrophy (DMD). The study will enroll boy…

This phase 3 trial tests whether givinostat, an oral drug, can slow muscle decline in boys aged 9 to 18 with Duchenne muscular dystrophy who can no longer walk. Participants receive either givinostat or a placebo for 18 months. The main goal is to see if the drug helps maintain u…

This study tests a single dose of SGT-003, a gene therapy that delivers a working version of the dystrophin gene to muscle cells. About 60 boys with Duchenne muscular dystrophy, aged from infancy to under 18, will receive the treatment and be followed for 5 years. The goal is to …

This study tests a single intravenous dose of a gene therapy called GNR-097 in boys aged 4 to 9 with Duchenne muscular dystrophy (DMD). The therapy uses a harmless virus to deliver a shortened version of the dystrophin gene, which is missing or faulty in DMD. The trial aims to se…

This study is testing whether a drug called tadalafil (often used for erectile dysfunction) combined with a home cycling program can help boys with Duchenne muscular dystrophy. The drug aims to improve blood flow to muscles, which is often poor in DMD, while exercise builds stren…

This early-phase trial tests whether injecting lab-grown muscle cells (MyoPAXon) into the foot is safe for adults with Duchenne muscular dystrophy who can no longer walk. Eight participants will receive the cells along with an immunosuppressant drug to prevent rejection. The main…

This program offers ongoing access to vamorolone, a steroid-like drug, for boys with Duchenne muscular dystrophy who finished earlier studies. It aims to keep them on treatment while the drug is being reviewed for approval. Participants take vamorolone daily by mouth at doses cho…

This study looks at the long-term safety and effects of the drug givinostat in people with Duchenne muscular dystrophy (DMD). It is open to those who have already taken part in a previous givinostat study. The goal is to track side effects and see how well the drug works over tim…

This pilot study tests whether VM100, a nutritional supplement made from ten natural ingredients, can improve quality of life for people with Duchenne muscular dystrophy (DMD). About 20 participants aged 8 and older will take VM100 or a placebo for 10 weeks. Researchers will meas…

This study looks at whether the Alexander technique, a method that teaches better posture and movement habits, can help children aged 5 to 9 with Duchenne muscular dystrophy control their upper limbs and feel better day-to-day. About 33 kids will take part, and researchers will m…

This study tests whether a fun, game-based occupational therapy program can improve quality of life, daily skills, and emotional health in 20 teens with Duchenne muscular dystrophy. Participants will use a computer or tablet at home. Researchers will compare results to standard t…

This study tests a new home-based program that uses a special treadmill and virtual reality to help children with conditions like cerebral palsy, muscle diseases, or obesity improve their walking. About 30 children aged 6 to 17 will try the program at home for at least 3 sessions…

Researchers in France are studying how different mutations in the DMD gene relate to brain, vision, and thinking differences in boys with dystrophinopathies. The study enrolls 110 children aged 5 to 12 who have a confirmed DMD diagnosis. Participants undergo sensory and neuropsyc…

This study will collect electronic health records from up to 2500 people with Duchenne or Becker muscular dystrophy, including female carriers, across U.S. clinics. The data will be combined with patient-reported information to give researchers a fuller picture of the diseases. T…

This study uses three wearable devices—a continuous glucose monitor, a heart monitor, and an activity tracker—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 DMD. The goal…

This study is building a worldwide 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 disease and to match patients with clinical trials. The goal i…

This study follows 220 boys aged 4 to 9 with Duchenne muscular dystrophy (DMD) for up to 3 years. Researchers collect data on muscle strength, walking ability, heart and lung function, and quality of life to better understand how the disease progresses. The goal is to gather natu…

This study aims to find less invasive ways to measure Duchenne muscular dystrophy activity. Researchers will collect urine and blood samples from 100 participants, including boys with Duchenne or Becker muscular dystrophy and healthy adults. They will look for RNA biomarkers that…

This study aims to find less invasive ways to measure muscle disease activity in people with muscular dystrophies. Instead of painful muscle biopsies, researchers will use blood and urine samples along with painless ultrasound and electrical tests on the arms and legs. The goal i…

This study will test if wearable devices like continuous glucose monitors and heart rate monitors can help detect early signs of heart problems in people with Duchenne muscular dystrophy (DMD). Ten participants will wear these devices at home over two years and have periodic hear…

This study uses MRI scans to track muscle damage and fat buildup in boys and men with Duchenne or Becker muscular dystrophy over 5-10 years. Researchers will compare these images with how well participants can walk and perform daily activities. The goal is to find better ways to …