Muscular dystrophy
MONDO:0020121Muscular dystrophy (MD) refers to a group of more than 30 genetic diseases characterized by progressive weakness and degeneration of the skeletal muscles that control movement. Some forms of MD are seen in newborns, infants or children, while others have late-onset and may not appear until middle age or later. The disorders differ in terms of the distribution and extent of muscle weakness (some forms of MD also affect cardiac muscle), age of onset, rate of progression, and pattern of inheritance. The prognosis for people with MD varies according to the type and progression of the disorder. There is no specific treatment to stop or reverse any form of MD. Treatment is supportive and may include physical therapy, respiratory therapy, speech therapy, orthopedic appliances used for support, corrective orthopedic surgery, and medicationsincluding corticosteroids, anticonvulsants (seizure medications), immunosuppressants, and antibiotics. Some individuals may need assisted ventilation to treat respiratory muscle weaknessor a pacemaker for cardiac (heart)abnormalities.
290 clinical trials for this condition and its sub-types, 74 tagged with Muscular dystrophy itself.
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Sub-types of Muscular dystrophy
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DMD-related muscular dystrophy 0 trials · 146 incl. sub-types
2 sub-types
- Duchenne muscular dystrophy 145 trials
- Becker muscular dystrophy 23 trials Sub-types →
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Progressive muscular dystrophy 2 trials · 125 incl. sub-types
13 sub-types
- Myotonic dystrophy 56 trials · 57 incl. sub-types Sub-types →
- Facioscapulohumeral muscular dystrophy 36 trials · 40 incl. sub-types Sub-types →
- Limb-girdle muscular dystrophy 17 trials · 26 incl. sub-types Sub-types →
- Emery-Dreifuss muscular dystrophy 2 trials · 4 incl. sub-types Sub-types →
- Symptomatic form of muscular dystrophy of Duchenne and Becker in female carriers 4 trials
- Oculopharyngeal muscular dystrophy 3 trials Sub-types →
- Congenital fibrosis of extraocular muscles 1 trial Sub-types →
- Bethlem myopathy 0 trials Sub-types →
- X-linked myopathy with excessive autophagy 0 trials Sub-types →
- Childhood-onset progressive contractures-limb-girdle weakness-muscle dystrophy syndrome 0 trials
- Myopathy, myofibrillar, 9, with early respiratory failure 0 trials
- Oculopharyngodistal myopathy 0 trials Sub-types →
- Progressive scapulohumeroperoneal distal myopathy 0 trials
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Congenital muscular dystrophy 1 trial · 10 incl. sub-types
23 sub-types
- Congenital merosin-deficient muscular dystrophy 1A 3 trials
- Congenital myasthenic syndrome 10 3 trials
- Congenital muscular dystrophy due to LMNA mutation 2 trials
- Muscular dystrophy-dystroglycanopathy 0 trials · 1 incl. sub-types Sub-types →
- Rigid spine syndrome 0 trials · 1 incl. sub-types Sub-types →
- Bethlem myopathy 0 trials Sub-types →
- SNUPN-related muscular dystrophy with or without multi-system involvement 0 trials Sub-types →
- Ullrich congenital muscular dystrophy 0 trials Sub-types →
- Arthrogryposis due to muscular dystrophy 0 trials
- Autosomal recessive myogenic arthrogryposis multiplex congenita 0 trials
- Collagen 6-related congenital muscular dystrophy 0 trials Sub-types →
- Congenital muscular dystrophy 1B 0 trials
- Congenital muscular dystrophy caused by variation in POMGNT2 0 trials Sub-types →
- Congenital muscular dystrophy due to integrin alpha-7 deficiency 0 trials
- Congenital muscular dystrophy with cataracts and intellectual disability 0 trials
- Congenital muscular dystrophy with hyperlaxity 0 trials
- Congenital muscular dystrophy without intellectual disability 0 trials
- Congenital muscular dystrophy-infantile cataract-hypogonadism syndrome 0 trials
- Congenital muscular dystrophy-respiratory failure-skin abnormalities-joint hyperlaxity syndrome 0 trials
- Congenital myopathy, Paradas type 0 trials
- Megaconial type congenital muscular dystrophy 0 trials
- Muscle-eye-brain disease 0 trials Sub-types →
- Muscular dystrophy, congenital, with rapid progression 0 trials
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Distal myopathy 1 trial · 4 incl. sub-types
11 sub-types
- Miyoshi myopathy 1 trial · 2 incl. sub-types Sub-types →
- Myopathy, distal, 5 1 trial
- MYH7-related skeletal myopathy 0 trials
- Asymptomatic hyperckemia-myalgia-rhabdomyolysis syndrome 0 trials
- Autosomal dominant distal myopathy 0 trials Sub-types →
- Distal myopathy with anterior tibial onset 0 trials
- Myopathy, distal, 7, adult-onset, X-linked 0 trials
- Myopathy, distal, infantile-onset 0 trials
- Myopathy, distal, with rimmed vacuoles 0 trials
- Nebulin-related early-onset distal myopathy 0 trials
- Oculopharyngodistal myopathy 0 trials Sub-types →
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LAMA2-related muscular dystrophy 2 trials · 3 incl. sub-types
2 sub-types
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Fukuda-Miyanomae-Nakata syndrome 0 trials
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Muscular dystrophy, Barnes type 0 trials
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Muscular dystrophy, Mabry type 0 trials
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Muscular dystrophy, cardiac type 0 trials
Most studied deeper sub-types
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One shot of gene therapy aims to fix a rare Muscle-Weakening disease
Cure Not yet recruitingResearchers are testing AMP-101, an experimental gene therapy, in people with DOK7 congenital myasthenic syndrome, a rare inherited disorder that causes muscle weakness and exercise intolerance. About 4 participants with moderate to severe DOK7 CMS will receive a single intraveno…
Phase 1 • Sponsor: Amplo Biotechnology • Aim: Cure
Last updated Sep 21, 2026 21:00 UTC
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Can an experimental infusion slow muscle loss in FSHD?
Disease control Not yet recruitingThis trial tests an experimental drug called SFL-0821 in adults with facioscapulohumeral muscular dystrophy (FSHD), a genetic condition that causes progressive muscle weakness. Researchers give the drug by intravenous infusion, either once or multiple times, and compare it agains…
Phase 1/2 • Sponsor: Soufflé Therapeutics, Inc. • Aim: Disease control
Last updated Sep 20, 2026 00:00 UTC
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New study aims to make duchenne gene therapy safer for kids
Disease control Not yet recruitingThis 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…
Phase 4 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated Aug 29, 2026 00:00 UTC
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Can a new dosing schedule tame steroid side effects in duchenne?
Disease control Not yet recruitingThis trial tests two ways of taking the steroid prednisolone in boys aged 4-10 with Duchenne muscular dystrophy who can still walk. One group takes the drug every other day, while the other takes it for 10 days on, then 10 days off. The goal is to see which schedule better preser…
Sponsor: Muhammad Aamir Latif • Aim: Disease control
Last updated Aug 28, 2026 00:00 UTC
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Experimental drug KER-065 aims to slow muscle decline in duchenne muscular dystrophy
Disease control Not yet recruitingThis 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…
Phase 2 • Sponsor: Keros Therapeutics, Inc. • Aim: Disease control
Last updated Aug 12, 2026 00:00 UTC
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Mind-Controlled computers: new device aims to help paralyzed veterans regain independence
Disease control Not yet recruitingThis study will test a mobile brain-computer interface (iBCI) that lets people with severe paralysis control computers and mobile devices using only their thoughts. Two veterans with conditions like spinal cord injury or ALS will use the device at home. The goal is to see if the …
Sponsor: VA Office of Research and Development • Aim: Disease control
Last updated Jul 29, 2026 00:00 UTC
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New drugs aim to slow muscle decline in duchenne muscular dystrophy
Disease control Not yet recruitingThis study tests two experimental drugs, ENTR-601-44 and ENTR-601-45, in people with Duchenne muscular dystrophy (DMD) who have already taken part in earlier studies. The goal is to see if long-term use is safe and tolerable, and whether it can help maintain muscle function. Part…
Phase 2 • Sponsor: Entrada Therapeutics, Inc. • Aim: Disease control
Last updated Jul 04, 2026 00:00 UTC
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Could a popular Weight-Loss drug help—or Harm—Those with duchenne muscular dystrophy?
Disease control Not yet recruitingThis study tests whether semaglutide (a GLP-1 drug used for weight loss) is safe for adolescents and young adults with Duchenne muscular dystrophy (DMD) who are also overweight or obese. The main concern is that the drug might reduce muscle mass, which is already weak in DMD. Par…
Phase 1/2 • Sponsor: Vanderbilt University Medical Center • Aim: Disease control
Last updated Jun 27, 2026 13:07 UTC
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New hope for DMD? small study tests immune drug to slow muscle decline
Disease control Not yet recruitingThis study tests the safety of a drug called rituximab in 5 boys aged 6-10 with Duchenne muscular dystrophy (DMD) who can still walk. The drug aims to lower certain immune proteins to possibly slow muscle damage. Researchers will monitor side effects and measure changes in walkin…
Sponsor: Peking Union Medical College Hospital • Aim: Disease control
Last updated Jun 27, 2026 11:02 UTC
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Can a Three-Hour online class help families manage bladder and bowel issues in duchenne muscular dystrophy?
Symptom relief Not yet recruitingResearchers are testing whether a single online urotherapy education session can improve parents' knowledge and daily habits around bladder and bowel care for children with Duchenne muscular dystrophy. About 50 parents or caregivers will take part. The session covers urinary trac…
Sponsor: Lokman Hekim University • Aim: Symptom relief
Last updated Sep 17, 2026 00:00 UTC
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Exercise hope: new study tests Muscle-Strengthening in kids with DMD
Symptom relief Not yet recruitingThis study will test different types of strengthening exercises in 45 children aged 6 to 10 with Duchenne muscular dystrophy (DMD). The goal is to see if these exercises can improve muscle strength, walking distance, and chemical markers in the blood. The results could help guide…
Sponsor: Cairo University • Aim: Symptom relief
Last updated Jun 27, 2026 12:32 UTC
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Video game physio for duchenne kids: fun or flop?
Symptom relief Not yet recruitingThis study tests whether a gamified mobile app can help 46 boys aged 6–12 with Duchenne muscular dystrophy stay motivated with their home physiotherapy. The app includes rewards, levels, and feedback to make exercise more engaging. Researchers will measure physical function, endu…
Sponsor: Başak Çağla Arslan • Aim: Symptom relief
Last updated Jun 27, 2026 09:00 UTC
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Can a support group boost confidence for parents of kids with DMD or SMA?
Symptom relief Not yet recruitingThis study tests whether a multicomponent support group can improve the confidence (self-efficacy) of primary caregivers of children with Duchenne muscular dystrophy or spinal muscular atrophy in Pakistan. Thirty caregivers will join group sessions with doctors, therapists, and o…
Sponsor: Aga Khan University • Aim: Symptom relief
Last updated Jun 27, 2026 08:12 UTC
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Could a common blood pressure pill ease muscle stiffness? early trial launches
Symptom relief Not yet recruitingThis early-stage trial tests amlodipine, a calcium channel blocker used for high blood pressure, in 20 adults with myotonic dystrophy type 1. The goal is to see if the drug is safe and can improve muscle strength, reduce stiffness, and help with daily function. All participants w…
Phase 1 • Sponsor: University of Rochester • Aim: Symptom relief
Last updated Jun 27, 2026 08:03 UTC
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Can brain scans unlock hidden effects of becker muscular dystrophy?
Knowledge-focused Not yet recruitingThis study looks at how the absence of a protein called Dp140 in people with Becker muscular dystrophy might affect the brain, thinking, and muscle function. Researchers will use brain and muscle imaging, along with cognitive and psychological tests, to explore these connections.…
Sponsor: Institut de Myologie, France • Aim: Knowledge-focused
Last updated Aug 02, 2026 00:00 UTC
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Can watching the course of myotonic dystrophy unlock better care?
Knowledge-focused Not yet recruitingThis study follows 100 adults with myotonic dystrophy (types 1 or 2) for two years to understand how muscle stiffness, daily function, and heart health change over time. Researchers will look back at up to 18 months of past medical records and then track participants with clinic …
Sponsor: Lupin Ltd. • Aim: Knowledge-focused
Last updated Jul 30, 2026 00:00 UTC
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Wearable sensors could bring duchenne monitoring home
Knowledge-focused Not yet recruitingThis study tests whether a wearable sensor (Syde device) can reliably measure arm and hand function in boys with Duchenne muscular dystrophy who can no longer walk. Current tests are done in clinics and may miss small changes. The goal is to see if home-based digital monitoring i…
Sponsor: Centre Hospitalier Universitaire de Liege • Aim: Knowledge-focused
Last updated Jun 27, 2026 14:02 UTC
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500 kids with muscular dystrophy to be tracked in major new study
Knowledge-focused Not yet recruitingThis study will follow 500 boys aged 1 to 18 with Duchenne or Becker muscular dystrophy for several years. Researchers will track their muscle function, body composition, lab tests, and heart and lung imaging to map how the disease changes over time. The goal is to create a model…
Sponsor: West China Second University Hospital • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:04 UTC
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New registry aims to improve care for muscular dystrophy patients
Knowledge-focused Not yet recruitingThis study is creating a registry for people with Duchenne and Becker muscular dystrophy, as well as symptomatic female carriers. The goal is to collect health data and quality-of-life information to monitor how new therapies work in real-world settings. Up to 1,500 participants …
Sponsor: Dr. Andreas Ziegler • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:05 UTC
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New study seeks to unlock the secrets of physical activity in kids with DMD
Knowledge-focused Not yet recruitingThis study aims to identify the many factors that influence how physically active children with Duchenne Muscular Dystrophy (DMD) are. Researchers will look at muscle strength, balance, thinking skills, sleep, behavior, and family environment. The goal is to gather information th…
Sponsor: Hacettepe University • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:00 UTC