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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Can a brain implant let paralyzed people control computers with their minds?
Disease control OngoingThis study tests a device called a motor neuroprosthesis, an implantable brain computer interface designed to help people with severe paralysis control digital devices like computers or tablets. The device aims to bypass damaged motor pathways and transmit brain signals directly …
Sponsor: Synchron Medical, Inc. • Aim: Disease control
Last updated Sep 10, 2026 00:00 UTC
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Heart cell therapy shows promise for duchenne MD in major trial
Disease control OngoingThis Phase 3 trial tests a cell therapy called deramiocel (CAP-1002) in 106 boys and young men with Duchenne muscular dystrophy. Participants receive either the cell therapy or a placebo every 3 months for a year, then all can receive the therapy for another year. The goal is to …
Phase 3 • Sponsor: Capricor Inc. • Aim: Disease control
Last updated Aug 30, 2026 00:00 UTC
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Gene therapy trial offers hope for rare muscle disease
Disease control OngoingThis study tests a single dose of a gene therapy called AB-1003 in 10 adults with a rare genetic muscle disease (LGMD2I/R9). The goal is to see if it is safe and can help improve muscle function. Participants must be able to walk or run 10 meters in under 30 seconds.
Phase 1/2 • Sponsor: AskBio Inc • Aim: Disease control
Last updated Aug 28, 2026 00:00 UTC
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New hope for muscle disease: experimental drug VX-670 enters human trials
Disease control OngoingThis early-stage trial tests the safety and tolerability of a new drug called VX-670 in 52 adults with myotonic dystrophy type 1 (DM1), a genetic condition that causes muscle weakness and other problems. Participants receive either VX-670 or a placebo, and researchers will monito…
Phase 1/2 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated Aug 22, 2026 00:00 UTC
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Experimental gene 'Silencer' therapy enters human testing for rare muscle disease
Disease control OngoingThis early-phase trial is testing EPI-321, a one-time gene therapy designed to silence the faulty gene that causes facioscapulohumeral muscular dystrophy (FSHD). The study will enroll 12 adults with FSHD Type 1 to see if the treatment is safe and tolerable, and whether it shows a…
Phase 1/2 • Sponsor: Epicrispr Biotechnologies, Inc. • Aim: Disease control
Last updated Aug 21, 2026 00:00 UTC
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Vertex tests long-term safety of VX-670 for muscle disease
Disease control By invitation onlyThis study tests the long-term safety and effectiveness of an experimental drug called VX-670 in adults with myotonic dystrophy type 1 (DM1). Participants who completed a previous VX-670 study can join. The drug is given through a vein, and researchers will monitor side effects a…
Phase 2 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated Aug 12, 2026 00:00 UTC
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New hope for FSHD: Long-Term drug safety trial underway
Disease control OngoingThis study is for people with FSHD, a genetic disease that causes muscle weakness. It tests the long-term safety and how well the body tolerates a drug called AOC 1020, given through a vein. About 84 adults who completed a previous study will take part. The main goal is to check …
Phase 2 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated Jul 16, 2026 00:00 UTC
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Hope for duchenne: new cell therapy shows promise in Long-Term trial
Disease control OngoingThis study tests the long-term safety and effectiveness of a cell therapy called deramiocel (CAP-1002) in people with Duchenne muscular dystrophy who completed the earlier HOPE-2 trial. Participants receive an infusion of deramiocel every 3 months for about 5 years, with the opti…
Phase 2 • Sponsor: Capricor Inc. • Aim: Disease control
Last updated Jun 27, 2026 07:56 UTC
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Desperate hope: stem cells tested on one child with rare muscle disease
Disease control Expanded access (ended)This trial gives a single child with a rare form of congenital muscular dystrophy access to their own banked stem cells. The cells are given through 14 IV infusions to see if they are safe and can help control the disease. Because it involves only one patient, the results will be…
Sponsor: Hope Biosciences Research Foundation • Aim: Disease control
Last updated Jun 27, 2026 07:52 UTC
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New tools aim to speed up FSHD drug trials
Knowledge-focused OngoingThis study involves 324 adults with facioscapulohumeral muscular dystrophy (FSHD) across 14 international sites. Researchers are testing two new measurement tools—a functional test called FSHD-COM and a muscle-impedance device—to better track disease progression. The goal is to i…
Sponsor: University of Kansas Medical Center • Aim: Knowledge-focused
Last updated Sep 19, 2026 00:00 UTC
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Massive study aims to unlock secrets of childhood nerve and muscle diseases
Knowledge-focused OngoingThis long-term study looks at children and adults with inherited nerve and muscle disorders that start early in life, like muscular dystrophy. Researchers will track symptoms over time and collect genetic samples from affected individuals, their family members, and healthy volunt…
Sponsor: National Institute of Neurological Disorders and Stroke (NINDS) • Aim: Knowledge-focused
Last updated Sep 11, 2026 00:00 UTC
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New study aims to sharpen tools for tracking becker muscular dystrophy
Knowledge-focused OngoingThis 24-month observational study will follow 80 people with Becker muscular dystrophy (BMD) to better understand how the disease progresses. Researchers will measure muscle strength, walking speed, breathing, and heart function using standard tests. The goal is to identify which…
Sponsor: Virginia Commonwealth University • Aim: Knowledge-focused
Last updated Sep 05, 2026 00:00 UTC
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Brain scans aim to unlock mysteries of muscle disease
Knowledge-focused By invitation onlyThis study looks at how myotonic dystrophy types 1 and 2 affect the brain. About 100 adults aged 30-65 will have MRI scans, thinking and movement tests, and blood draws. Some will also have a spinal tap. The goal is to find brain changes that could be used as markers in future tr…
Sponsor: Wake Forest University Health Sciences • Aim: Knowledge-focused
Last updated Jul 16, 2026 00:00 UTC
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New model aims to speed up rare disease diagnosis
Knowledge-focused OngoingThis study is testing a new way to care for people with rare diseases. It will use advanced genetic testing and a team of specialists to help diagnose patients faster and coordinate their care better. The study involves 136 participants with certain rare diseases and aims to redu…
Sponsor: Fondazione Policlinico Universitario Agostino Gemelli IRCCS • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:25 UTC
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Scientists build heart cells in a dish to unravel genetic heart disease
Knowledge-focused By invitation onlyThis study collects blood or skin samples from 100 adults with inherited heart rhythm disorders (like Long QT Syndrome or Brugada Syndrome) and healthy volunteers. Researchers will turn these samples into stem cells and then into heart cells to study how these diseases work and t…
Sponsor: Johns Hopkins University • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:11 UTC
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New study aims to speed up FSHD drug development
Knowledge-focused OngoingThis study is working to create better tools for measuring muscle function in people with facioscapulohumeral muscular dystrophy (FSHD). Researchers will follow 100 adults with FSHD for up to 24 months, testing new ways to track disease progression. The goal is to make future cli…
Sponsor: Centre Hospitalier Universitaire de Nice • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:01 UTC
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Scientists watch LGMD progress in 205 patients over years
Knowledge-focused OngoingThis study follows 205 people with four types of limb-girdle muscular dystrophy (LGMD) to understand how the disease changes over time. Participants will have their muscle strength, movement, and breathing tested regularly for up to 5 years. No treatment is given; the goal is to …
Sponsor: Sarepta Therapeutics, Inc. • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:56 UTC