DUCHENNE MUSCULAR DYSTROPHY

This program provides continued access to the drug vamorolone for boys with Duchenne muscular dystrophy (DMD) who have already taken it in previous clinical studies. It allows them to keep receiving the medication while it is being reviewed for full market approval. The goal is t…

This study is testing whether a medication called satralizumab, given as a shot every four weeks, can improve bone strength and muscle function in children with Duchenne muscular dystrophy. The trial includes 50 boys aged 8 to 18 who are already taking daily steroid medication. R…

This study is testing whether a single intravenous dose of an experimental gene therapy called SGT-003 can help boys with Duchenne muscular dystrophy (DMD) walk and move better. It will involve 80 boys who can still walk, comparing those who get the therapy to those who get a pla…

This study is testing a new oral medicine called SAT-3247 for boys aged 7 to under 10 who have Duchenne Muscular Dystrophy (DMD) and can still walk. The main goals are to find a safe and well-tolerated dose and to see if the medicine can help improve muscle strength and function.…

This study aims to understand the long-term safety and effectiveness of the drug GIVINOSTAT in boys with Duchenne muscular dystrophy (DMD). It is open to about 206 participants, aged 6 and older, who have already taken GIVINOSTAT in a previous related study. The main goal is to m…

This is an early safety study testing a single spinal injection of an experimental gene therapy called INS1201. It involves up to 12 young boys, aged 2 to under 5 years old, who have Duchenne muscular dystrophy and can still walk. The main goal is to see if the treatment is safe …

This study is testing an investigational drug called WVE-N531 for boys with Duchenne muscular dystrophy (DMD) who have a specific genetic mutation. The main goal is to see if the drug is safe and if it can increase levels of a crucial muscle protein called dystrophin. Researchers…

This study is testing a new drug called NS-089/NCNP-02 for boys with Duchenne muscular dystrophy (DMD) caused by a specific genetic error (exon 44). The drug is given as a weekly IV infusion and aims to help muscles produce more of a crucial protein called dystrophin. Researchers…

This early-stage study is testing whether lab-grown muscle stem cells are safe when injected into the foot muscles of adults with Duchenne muscular dystrophy (DMD). Researchers will give eight non-walking adults a single injection and monitor them closely for side effects. The ma…

This study is testing a one-time intravenous gene therapy called RGX-202 for boys with Duchenne muscular dystrophy (DMD). The goal is to see if delivering a new, functional gene to muscles is safe and can help slow or stop the progression of the disease. The study will enroll abo…

This is the first study in humans of a new drug called NS-050/NCNP-03 for boys with Duchenne muscular dystrophy (DMD). The main goals are to check if the weekly injections are safe and to see how the drug moves through the body. Researchers will also measure if the treatment help…

This study is testing a single intravenous dose of an experimental gene therapy called SGT-003 in boys with Duchenne muscular dystrophy (DMD). The main goals are to see if the treatment is safe and if it helps produce a needed muscle protein (microdystrophin) and improves muscle …

This study aims to collect long-term safety information on the drug AGAMREE® (vamorolone) for people with Duchenne muscular dystrophy (DMD). It will follow about 250 male patients, aged 2 and older, for approximately 5 years to monitor their health and see how the treatment affec…

This early-stage study is testing a new drug called BMN 351 in boys with Duchenne muscular dystrophy (DMD), a serious muscle-wasting condition. The main goal is to find out if the drug is safe and how the body processes it when given through an IV. The study will involve about 18…

This study is testing if making occupational therapy more like a video game can help teenagers with Duchenne muscular dystrophy. It aims to improve their ability to do daily tasks, their mood, and their overall quality of life compared to standard therapy. The research will invol…

This study aims to find easier ways to measure muscular dystrophy disease activity. Researchers will collect urine and blood samples and use painless scans on the arms and legs of about 465 participants. The goal is to see if these simple tests can work as well as or better than …

This study aims to track how Duchenne Muscular Dystrophy (DMD) naturally progresses in boys aged 4 to 9 years old over 6 to 36 months. Researchers will measure muscle function, mobility, heart health, and breathing to establish baseline data. This information will help design and…

This study aims to understand how many males with Duchenne muscular dystrophy (DMD) have certain antibodies in their blood. Researchers are screening up to 200 males aged 0 to 25 with DMD. The information will help identify who might be eligible for future gene therapy clinical t…

This study aims to find easier ways to measure how Duchenne muscular dystrophy is progressing. Researchers want to see if they can detect signs of the disease in urine and blood samples instead of needing painful muscle tissue biopsies. They are enrolling 100 males with Duchenne …

This study aims to develop better ways to track muscular dystrophy progression using MRI scans instead of invasive tests. Researchers will follow 550 boys and men with Duchenne or Becker muscular dystrophy for 5-10 years, comparing their muscle scans to healthy individuals. The g…

This study aims to understand if high blood sugar and changes in heart rate patterns are linked to heart problems in people with Duchenne Muscular Dystrophy (DMD). Researchers will use wearable devices like glucose monitors and heart monitors on 10 male participants over two year…