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.
288 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 · 124 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 →
- Symptomatic form of muscular dystrophy of Duchenne and Becker in female carriers 4 trials
- Emery-Dreifuss muscular dystrophy 1 trial · 3 incl. sub-types Sub-types →
- 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 · 9 incl. sub-types
23 sub-types
- Congenital merosin-deficient muscular dystrophy 1A 3 trials
- Congenital muscular dystrophy due to LMNA mutation 2 trials
- Congenital myasthenic syndrome 10 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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DMD drug tested in wheelchair users – but trial stops early
Disease control Stopped earlyThis study tested the safety of golodirsen (Vyondys 53) in boys and men with Duchenne muscular dystrophy who can no longer walk. Only 2 people took part before the trial was stopped early. Participants received weekly IV infusions for up to 96 weeks, with extra follow-up. The goa…
Phase 4 • Sponsor: Rare Disease Research, LLC • Aim: Disease control
Last updated Jun 27, 2026 12:03 UTC
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Experimental gene therapy for rare muscle disease shows early promise but study halted
Disease control Stopped earlyThis study tested a gene therapy called SRP-9003 for people with limb-girdle muscular dystrophy type 2E (LGMD2E), a rare genetic disease that causes muscle weakness. The treatment aimed to deliver a working gene to muscle cells to help them produce a missing protein. Only 6 peopl…
Phase 1/2 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated Jun 27, 2026 08:12 UTC
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Experimental drug losmapimod tested in rare muscle disease – early hopes, but trial cut short
Disease control Stopped earlyThis study tested an experimental drug called losmapimod in 14 adults with FSHD1, a rare genetic condition that causes progressive muscle weakness. The main goal was to check safety and tolerability, and to see if the drug affects certain biological markers. The trial was termina…
Phase 2 • Sponsor: Fulcrum Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 08:01 UTC
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New drug trial for duchenne MD halted early – what we know
Disease control Stopped earlyThis phase 2 study tested a drug called PGN-EDO51 in 7 people with Duchenne muscular dystrophy whose genetic mutation can be corrected by skipping exon 51. The drug was given by IV infusion to see if it is safe and tolerable. The trial was terminated, so results are limited.
Phase 2 • Sponsor: PepGen Inc • Aim: Disease control
Last updated Jun 27, 2026 08:01 UTC
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Experimental gene therapy for DMD hits antibody barrier – study halted
Disease control Stopped earlyThis study tested a gene therapy (delandistrogene moxeparvovec) combined with a drug called imlifidase to see if it could safely deliver the therapy to boys with Duchenne muscular dystrophy who had antibodies that might block the treatment. Only 5 participants were planned, but t…
Phase 1 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated Jun 27, 2026 07:59 UTC
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FSHD drug trial halted midway: what happened?
Disease control Stopped earlyThis study tested a drug called losmapimod for people with a rare muscle-weakening disease called FSHD. The goal was to see if the drug could slow muscle loss and improve arm function over 48 weeks. About 260 adults with FSHD were randomly assigned to receive either losmapimod or…
Phase 3 • Sponsor: Fulcrum Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 07:56 UTC
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Experimental gene therapy targets duchenne MD in young boys
Disease control Stopped earlyThis Phase 2 trial tested a single dose of gene therapy (fordadistrogene movaparvovec) in 10 boys with early-stage Duchenne muscular dystrophy. The goal was to check safety and whether the therapy could help muscles produce a mini-dystrophin protein. The study was terminated earl…
Phase 2 • Sponsor: Pfizer • Aim: Disease control
Last updated Jun 27, 2026 07:55 UTC
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Gene therapy for duchenne muscular dystrophy under Long-Term watch
Disease control Stopped earlyThis study follows 7 people with Duchenne muscular dystrophy who previously received an experimental gene therapy called fordadistrogene movaparvovec. Researchers will monitor them for 10 years to check for side effects and see if the treatment continues to help with movement. Th…
Phase 3 • Sponsor: Pfizer • Aim: Disease control
Last updated Jun 27, 2026 07:55 UTC
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Experimental drug losmapimod tested for rare muscle disease
Disease control Stopped earlyThis phase 2 trial tested the drug losmapimod in 76 adults with FSHD, a genetic condition that causes progressive muscle weakness. Participants took either losmapimod or a placebo for 48 weeks to see if the drug was safe and could help control the disease. The study was terminate…
Phase 2 • Sponsor: Fulcrum Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 07:52 UTC
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Gene therapy trial for duchenne MD halted early – what we know
Disease control Stopped earlyThis early-stage trial tested a single infusion of gene therapy (PF-06939926) in 23 people with Duchenne muscular dystrophy, both those who could still walk and those who could not. The main goal was to check safety and tolerability, while also measuring dystrophin protein levels…
Phase 1 • Sponsor: Pfizer • Aim: Disease control
Last updated Jun 26, 2026 17:12 UTC
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Can plasma exchange clear the way for gene therapy in duchenne?
Disease control Stopped earlyThis early study tested whether a blood-cleaning procedure called plasmapheresis could allow boys with Duchenne muscular dystrophy who have antibodies against the gene therapy carrier to still receive the treatment. Only 3 boys were enrolled before the study was stopped early. Th…
Phase 1 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated Jun 26, 2026 14:20 UTC
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Experimental gene therapy for rare muscle disease tested in just 2 people
Disease control Stopped earlyThis was a very early (Phase 1) study testing a gene therapy called SRP-6004 for people with limb girdle muscular dystrophy type 2B/R2, a rare muscle-weakening disease. The goal was to see if a single IV infusion of the therapy is safe and can help the body produce a missing prot…
Phase 1 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated Jun 26, 2026 14:20 UTC
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Experimental gene therapy tested for rare muscular dystrophy
Disease control Stopped earlyThis early-stage trial tested a gene therapy called SRP-9004 in just 4 people with limb girdle muscular dystrophy type 2D/R3, a rare muscle-weakening disease. The main goal was to check safety, not effectiveness. The study was terminated early, so results are limited.
Phase 1 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated Jun 26, 2026 13:47 UTC
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Gut hormone shot aims to strengthen fragile bones in kids with muscle diseases
Knowledge-focused Stopped earlyThis study tested whether two gut hormones, GIP and GLP-2, could reduce bone breakdown in children with spinal muscular atrophy, cerebral palsy, or Duchenne muscular dystrophy who use wheelchairs. Participants received a liquid meal and then either a hormone injection or a placeb…
Sponsor: University of Copenhagen • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:08 UTC
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One-Person study aims to unlock FSHD mysteries
Knowledge-focused Stopped earlyThis study looked at one person with facioscapulohumeral muscular dystrophy (FSHD) to better understand the disease. Researchers examined muscle tissue and checked for specific biomarkers. The goal was to learn more about how FSHD affects the body, not to test a treatment.
Sponsor: Nationwide Children's Hospital • Aim: Knowledge-focused
Last updated Jun 26, 2026 17:43 UTC