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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New Steroid-Like drug tested for muscle disease
Disease control CompletedThis 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…
Phase 2 • Sponsor: ReveraGen BioPharma, Inc. • Aim: Disease control
Last updated Sep 17, 2026 00:00 UTC
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Experimental drug AOC 1001 tested for Long-Term safety in rare muscle disease
Disease control CompletedThis study is a follow-up to an earlier trial, testing the long-term safety and effects of a drug called AOC 1001 in adults with myotonic dystrophy type 1 (DM1), a genetic muscle disease. 37 participants who completed the first study received multiple doses of AOC 1001 by IV infu…
Phase 2 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated Sep 13, 2026 00:00 UTC
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New hope for FSHD: experimental drug AOC 1020 completes early testing
Disease control CompletedThis study tested a new medicine called AOC 1020 in 90 adults with facioscapulohumeral muscular dystrophy (FSHD), a genetic condition that causes muscle weakness. The goal was to check if the drug is safe and how the body processes it. Participants received either the drug or a p…
Phase 1/2 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated Jul 18, 2026 00:00 UTC
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New drug aims to help boys with duchenne walk longer
Disease control CompletedThis 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…
Phase 3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 11:00 UTC
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New drug PGN-EDODM1 tested for muscle disease
Disease control CompletedThis early-stage trial tested a single dose of the drug PGN-EDODM1 in 24 adults with myotonic dystrophy type 1, a genetic muscle disorder. The main goal was to check safety and how the body handles the drug. The study is complete, but results are not yet available.
Phase 1 • Sponsor: PepGen Inc • Aim: Disease control
Last updated Jun 27, 2026 09:04 UTC
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New steroid hope for duchenne boys: safer muscle treatment?
Disease control CompletedThis 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…
Phase 2 • Sponsor: Santhera Pharmaceuticals • Aim: Disease control
Last updated Jun 27, 2026 07:55 UTC
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New adjustable brace shows promise for kids with crouch gait
Symptom relief CompletedThis study tested a new type of leg brace designed to help children with cerebral palsy and other movement disorders walk more easily. The brace was custom-made for each child and could be adjusted to provide different kinds of support. Researchers measured how well children walk…
Sponsor: National Institutes of Health Clinical Center (CC) • Aim: Symptom relief
Last updated Sep 18, 2026 00:00 UTC
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Robotic arm could help people with paralysis regain independence at home
Symptom relief CompletedThis study tests whether a wheelchair-mounted robotic arm called Jaco helps people with tetraplegia perform daily tasks like grasping and reaching at home. Participants use the device for two months after training. Researchers measure changes in performance and satisfaction with …
Sponsor: Association APPROCHE • Aim: Symptom relief
Last updated Jul 04, 2026 00:00 UTC
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Robotic leg device shows promise for helping muscle disease patients walk
Symptom relief CompletedThis study tested a powered leg exoskeleton (Keeogo) in 50 people with various muscle disorders to see if it is safe and helps them walk better. Participants performed walking tests with and without the device. The goal was to see if the device could improve walking distance and …
Sponsor: Institut de Myologie, France • Aim: Symptom relief
Last updated Jun 27, 2026 12:08 UTC
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Robotic exoskeleton shows promise for muscle disease patients
Symptom relief CompletedThis study tested a wearable robotic suit called MyoSuit that assists knee and hip movement in people with various muscle disorders. 32 participants used the device to perform walking tests, and researchers checked for safety and any immediate improvements in walking ability. The…
Sponsor: Institut de Myologie, France • Aim: Symptom relief
Last updated Jun 27, 2026 12:08 UTC
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Can a global patient registry unlock the secrets of a rare muscle disease?
Knowledge-focused CompletedThis study is building an international registry of people with GNE myopathy, an ultra-rare muscle disease that causes progressive weakness and often leads to wheelchair use. Participants will complete online questionnaires about their symptoms, medical history, quality of life, …
Sponsor: Newcastle University • Aim: Knowledge-focused
Last updated Aug 19, 2026 00:00 UTC
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Inflammation may be key in rare muscle disease
Knowledge-focused CompletedThis pilot study examined whether inflammation, measured by cytokines in the blood, plays a role in type 1 facioscapulohumeral muscular dystrophy (FSHD1). Researchers compared 20 FSHD1 patients with healthy controls to see if certain inflammatory markers are higher in the disease…
Sponsor: Centre Hospitalier Universitaire de Nice • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:00 UTC
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New study maps key tests for LGMD to speed up drug development
Knowledge-focused CompletedThis study involved 116 people with Limb Girdle Muscular Dystrophy (LGMD), a group of rare muscle-weakening disorders. Researchers measured how well participants could walk, move their arms, and breathe, and asked about their daily activities and overall health. The goal was to i…
Sponsor: Virginia Commonwealth University • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:09 UTC