DUCHENNE MUSCULAR DYSTROPHY (DMD)

This study tests an experimental drug called ENTR-601-45 in 24 people with Duchenne muscular dystrophy who have a specific genetic mutation (exon 45 skipping). The goal is to check the drug's safety, find the right dose, and see if it can help muscles work better. Participants re…

This early-phase study tests a gene therapy called SPOT-03 in 9 boys with Duchenne muscular dystrophy (DMD). The main goal is to see if the treatment is safe and tolerable. Researchers will also check if the therapy increases dystrophin protein levels in muscles, which is missing…

This study tests an investigational drug called ENTR-601-44 in 24 boys and young men with Duchenne muscular dystrophy (DMD) who have a specific genetic change (exon 44 skipping). The goal is to see if the drug is safe, tolerable, and can help improve muscle function compared to a…

This study tests whether playing virtual reality games can improve arm and hand function in people with Duchenne muscular dystrophy (DMD). 36 participants will either do standard physical therapy or the same therapy plus VR exercises using a Meta Quest 3 headset. The goal is to s…

This study looks at how different types of gene changes in the DMD gene are linked to heart problems in boys and young men (ages 2-24) with Duchenne or Becker muscular dystrophy. Researchers will use heart tests and blood samples to find early signs of heart issues. The goal is t…

This study watches how babies with Duchenne muscular dystrophy grow and develop during their first three years. Researchers will track motor skills like crawling and walking, as well as thinking and learning abilities. The goal is to better understand the earliest signs of the di…

This study looks back at the last 10 years to understand how many people in Denmark use a breathing machine at home through a tube in their throat. Researchers will examine which diseases lead to this need and how many patients survive one year after starting. The goal is to gath…

This study is testing whether a protein called titin, found in urine, can serve as a reliable marker of muscle injury in people with Duchenne or Becker muscular dystrophy. Researchers will measure titin levels before and after physical activity, both in the clinic and at home, us…

This study aims to find new ways to measure walking problems in children with Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA). About 106 participants will wear special insoles that track their steps and movement during daily activities and in clinic tests. The…

This study follows 100 children with Duchenne muscular dystrophy or spinal muscular atrophy, identified through newborn screening, to see how their motor skills develop over time. Children wear special sensor garments or ankle monitors at home to track movement. The goal is to fi…