DUCHENNE MUSCULAR DYSTROPHY (DMD)
Clinical trials for DUCHENNE MUSCULAR DYSTROPHY (DMD) explained in plain language.
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Gene therapy SPOT-03 hopes to restore muscle protein in boys with DMD
Disease control Recruiting nowThis early-phase study tests a new gene therapy called SPOT-03 in 9 boys aged 2 to 8 with Duchenne muscular dystrophy (DMD). The main goal is to see if the treatment is safe and tolerable. Researchers will also check if SPOT-03 can increase dystrophin, a key muscle protein missin…
Matched conditions: DUCHENNE MUSCULAR DYSTROPHY (DMD)
Phase: EARLY_PHASE1 • Sponsor: Shanghai Siponuoyin Biotechnology Co Ltd • Aim: Disease control
Last updated May 17, 2026 03:45 UTC
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New hope for duchenne: experimental drug aims to restore muscle protein
Disease control Recruiting nowThis study tests an investigational drug called ENTR-601-45 for people with Duchenne muscular dystrophy (DMD) who have a specific genetic type (exon 45 skipping). The goal is to check safety, find the right dose, and see if it helps muscle function. About 24 males aged 4 to 20 wi…
Matched conditions: DUCHENNE MUSCULAR DYSTROPHY (DMD)
Phase: PHASE1, PHASE2 • Sponsor: Entrada Therapeutics, Inc. • Aim: Disease control
Last updated May 15, 2026 11:55 UTC
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New drug hopes to slow duchenne muscle damage in kids and young adults
Disease control Recruiting nowThis study tests an investigational medicine called ENTR-601-44 for people with Duchenne muscular dystrophy (DMD) whose genetic mutation allows skipping of exon 44. The trial has two parts: first to find the safest dose, then to see if it improves muscle function compared to a pl…
Matched conditions: DUCHENNE MUSCULAR DYSTROPHY (DMD)
Phase: PHASE1, PHASE2 • Sponsor: Entrada Therapeutics, Inc. • Aim: Disease control
Last updated May 08, 2026 12:02 UTC
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Smart insoles could reveal hidden clues in rare muscle diseases
Knowledge-focused Recruiting nowThis 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…
Matched conditions: DUCHENNE MUSCULAR DYSTROPHY (DMD)
Sponsor: Columbia University • Aim: Knowledge-focused
Last updated May 17, 2026 04:00 UTC
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Urine test could revolutionize DMD drug trials
Knowledge-focused Recruiting nowThis study is looking at whether a protein called titin, found in urine, can be used as a marker for muscle injury in people with Duchenne or Becker muscular dystrophy. Researchers will measure titin levels before and after physical activity, and compare them to activity levels r…
Matched conditions: DUCHENNE MUSCULAR DYSTROPHY (DMD)
Phase: NA • Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated May 15, 2026 11:54 UTC
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New study follows babies with DMD from birth to age 3 to spot early clues
Knowledge-focused Recruiting nowThis study follows 105 boys with Duchenne muscular dystrophy (DMD) from birth to age 3 to understand how the disease affects early development. Researchers will measure motor and thinking skills using standard baby tests. The goal is to learn more about the earliest signs of DMD,…
Matched conditions: DUCHENNE MUSCULAR DYSTROPHY (DMD)
Sponsor: University of Rochester • Aim: Knowledge-focused
Last updated May 13, 2026 15:59 UTC
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Smart socks and ankle sensors could spot muscle disease early in babies
Knowledge-focused Recruiting nowThis 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…
Matched conditions: DUCHENNE MUSCULAR DYSTROPHY (DMD)
Phase: NA • Sponsor: Centre Hospitalier Universitaire de Liege • Aim: Knowledge-focused
Last updated May 11, 2026 20:38 UTC
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Danish study reveals trends in home ventilator use for neuromuscular patients
Knowledge-focused Recruiting nowThis study looks back at the past 10 years to understand how many people in Denmark use a breathing machine at home through a tube in their windpipe. Researchers will track which diseases lead to this need, such as ALS or spinal cord injury, and how many patients survive one year…
Matched conditions: DUCHENNE MUSCULAR DYSTROPHY (DMD)
Sponsor: Rigshospitalet, Denmark • Aim: Knowledge-focused
Last updated May 06, 2026 16:02 UTC
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Heart gene link explored in muscular dystrophy study
Knowledge-focused Recruiting nowThis study aims to understand how different genetic 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 study…
Matched conditions: DUCHENNE MUSCULAR DYSTROPHY (DMD)
Sponsor: Aristotle University Of Thessaloniki • Aim: Knowledge-focused
Last updated Apr 29, 2026 15:15 UTC