DMD-related muscular dystrophy

This trial tests whether metformin, a drug that improves insulin sensitivity, can help overweight children with neurological or neuromuscular conditions. These children often face mobility challenges that lead to a sedentary lifestyle and weight gain. The study compares six month…

This trial asks whether whole-body vibration or gravity-force stimulation can better improve postural stability in children with Duchenne muscular dystrophy (DMD). It will include 60 children aged 6 to 10 who can walk independently. The study compares two exercise-based approache…

This Phase 3 trial tests whether TAS-205, an oral drug, can improve movement and safety in people with Duchenne muscular dystrophy. It includes 104 participants, both those who can walk and those who cannot. The study compares TAS-205 to a placebo over 52 weeks.

This early-phase trial tests the safety and tolerability of a new drug called GRT6019 in 24 healthy men. Participants receive multiple doses over 4 weeks and are monitored for side effects and how the drug moves through the body. The goal is to see if GRT6019 is safe enough to st…

This study tested an oral drug called ifetroban in 46 males aged 7 and older with Duchenne muscular dystrophy (DMD). The goal was to see if the drug is safe and can help prevent or treat heart problems, which are a leading cause of death in DMD. Participants received either ifetr…

This study looked at whether giving a steroid called prednisone twice a week at a lower dose, along with exercise training, could help boys with Duchenne muscular dystrophy (DMD) maintain muscle strength and function. The goal was to find a treatment that works as well as daily s…

This study tested the safety of a drug called ataluren (also known as Translarna) in 270 people with Duchenne muscular dystrophy caused by a specific type of genetic mistake (nonsense mutation). Participants had already taken ataluren in earlier studies. The goal was to monitor s…

This study tested a drug called ataluren in 360 boys with Duchenne muscular dystrophy caused by a specific genetic mistake (nonsense mutation). The main goal was to see if ataluren could help them walk farther over 72 weeks compared to a placebo. All participants were also taking…

This study tested whether a personalized diet could improve nutrition and movement in children aged 4-8 with Duchenne muscular dystrophy (DMD). Nine children in Ankara, Turkey, received dietary counseling focused on energy, protein, calcium, and fluid needs. Researchers measured …

This study tested two drugs, casimersen and golodirsen, in 228 boys with Duchenne muscular dystrophy who have specific genetic mutations. The goal was to see if these drugs could slow muscle decline, measured by how fast they could climb four steps. Participants received either t…

This study tested a drug called vamorolone in 54 boys with Duchenne muscular dystrophy, aged 2 to under 4 years and 7 to under 18 years. The goal was to check safety and how the body processes the drug over 12 weeks. Researchers hope vamorolone may help control the disease with f…

This phase 2 trial tested vamorolone, a drug similar to steroids, against a placebo in 46 males with Becker muscular dystrophy. The goal was to check safety and tolerability over 24 weeks, while also looking for early signs of benefit. The study is complete, but results are not y…

This early-stage trial tested a single-dose gene therapy called scAAV9.U7.ACCA in 3 boys with Duchenne muscular dystrophy caused by a duplication of exon 2. The therapy was given through a vein and aimed to help muscle cells produce dystrophin, a protein missing in this disease. …

This study tested whether nebivolol, a beta-blocker heart drug, could prevent heart failure in 51 boys aged 10-15 with Duchenne muscular dystrophy. Participants had normal heart function at the start and were randomly given either nebivolol or a placebo. The trial found that nebi…

This study tests a special fabric shoulder brace designed to mimic muscle support for people with neuromuscular disorders like muscular dystrophy, SMA, and ALS. About 30 participants will wear the brace and perform arm tasks to see if it improves movement, strength, and daily fun…

This study tested whether virtual reality (using Xbox Kinect) or biofeedback training could improve motor function and balance in 24 children with neuromuscular diseases like Duchenne muscular dystrophy and spinal muscular atrophy. Participants were split into three groups: VR tr…

This study tested whether an interactive virtual reality (VR) system could help children aged 5–10 with Duchenne muscular dystrophy (DMD) stick to their physiotherapy exercises at home. Fourteen children used the VR system for 8 weeks, and researchers measured how often and how l…

This study looked at whether virtual reality exercises and whole-body vibration can improve muscle strength and balance in children with Duchenne muscular dystrophy (DMD). Fifty children aged 6 to 10 years who could still walk took part. The goal was to see if these fun, non-drug…

This study tested whether breathing muscle exercises done at home via video calls could improve lung function and quality of life in 40 children with Duchenne muscular dystrophy. The children, aged 5-17, used a device to strengthen their breathing muscles. Researchers measured ch…

This study measured calf muscle thickness and length in 26 children with Duchenne muscular dystrophy (ages 6-12) who could still walk. Researchers used ultrasound and motion analysis to see how muscle structure relates to walking and other movements. The goal was to better unders…

This study follows boys with Duchenne muscular dystrophy who have small mutations in their genes, a group that is less understood than others. Over one year, researchers will use muscle MRI, genetic tests, and motor and respiratory assessments to see how the disease progresses. T…

This project creates a central repository of medical records, genetic test results, and patient-reported information from people with Duchenne or Becker muscular dystrophy, as well as carriers. Anyone aged 4 weeks or older with a confirmed diagnosis can join. The goal is to provi…

This study looked at the extra costs that families of people with Duchenne muscular dystrophy (DMD) in France have to pay out of their own pockets. It included 28 patients of any age who had been diagnosed for at least six months. The researchers measured things like uninsured me…

This study tested whether doctors can accurately assess physical function and quality of life in children with Duchenne muscular dystrophy (DMD) through video calls. Twenty ambulatory patients aged 5-18 completed timed tests and questionnaires remotely. The goal was to see if tel…

This completed study looked at how muscle oxygen levels change during exercise in people with various neuromuscular diseases compared to healthy volunteers. Seventeen participants did a knee-extension exercise while a device measured muscle oxygen. The goal was to understand diff…

This completed study enrolled 150 people with Duchenne muscular dystrophy to create a video database of their movement abilities. Caregivers recorded children doing specific tasks at home using a secure app. The goal was to help validate a new tool called the Duchenne Video Asses…

This study followed 316 people with Duchenne muscular dystrophy who were taking Translarna (ataluren) as part of their normal medical care. The goal was to monitor side effects and see how well the drug works in everyday use. Researchers also checked if doctors and patients follo…

This early-stage trial tested a single dose of the study drug GRT6019 in 18 healthy men to check its safety and how the body processes it. The study also looked at whether food changes drug absorption. Results will guide future research for Duchenne muscular dystrophy, but this t…