Progressive muscular dystrophy
MONDO:0016106Also known as: progressive muscular dystrophy
129 clinical trials for this condition and its sub-types, 2 tagged with Progressive muscular dystrophy itself.
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Sub-types of Progressive muscular dystrophy
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Myotonic dystrophy 56 trials · 57 incl. sub-types
8 sub-types
- Myotonic dystrophy type 1 45 trials Sub-types →
- Myotonic dystrophy type 2 11 trials
- Congenital myotonic dystrophy 9 trials
- Childhood-onset Steinert myotonic dystrophy 2 trials
- Juvenile-onset Steinert myotonic dystrophy 2 trials
- Adult-onset Steinert myotonic dystrophy 0 trials
- Late-onset Steinert myotonic dystrophy 0 trials
- Myotonic cataract 0 trials
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Facioscapulohumeral muscular dystrophy 36 trials · 40 incl. sub-types
5 sub-types
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Limb-girdle muscular dystrophy 17 trials · 26 incl. sub-types
3 sub-types
- Autosomal recessive limb-girdle muscular dystrophy 0 trials · 18 incl. sub-types Sub-types →
- Muscular dystrophy, limb-girdle, autosomal dominant 0 trials · 4 incl. sub-types Sub-types →
- POMGNT2-related limb-girdle muscular dystrophy R24 1 trial
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Oculopharyngeal muscular dystrophy 3 trials
2 sub-types
- Oculopharyngeal muscular dystrophy 1 0 trials
- Oculopharyngeal muscular dystrophy 2 0 trials
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Emery-Dreifuss muscular dystrophy 1 trial · 3 incl. sub-types
4 sub-types
- Autosomal dominant Emery-Dreifuss muscular dystrophy 0 trials · 3 incl. sub-types Sub-types →
- Emery-Dreifuss muscular dystrophy 3, autosomal recessive 0 trials
- X-linked Emery-Dreifuss muscular dystrophy 0 trials Sub-types →
- Scapuloperoneal myopathy 0 trials Sub-types →
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8 sub-types
- Tukel syndrome 0 trials
- Congenital fibrosis of extraocular muscles type 1 0 trials
- Fibrosis of extraocular muscles, congenital, 2 0 trials
- Fibrosis of extraocular muscles, congenital, 3A, with or without extraocular involvement 0 trials
- Fibrosis of extraocular muscles, congenital, 3b 0 trials
- Fibrosis of extraocular muscles, congenital, 3c 0 trials
- Fibrosis of extraocular muscles, congenital, 5 0 trials
- Fibrosis of extraocular muscles, congenital, with synergistic divergence 0 trials
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Bethlem myopathy 0 trials
4 sub-types
- Bethlem myopathy 1A 0 trials
- Bethlem myopathy 1B 0 trials
- Bethlem myopathy 1C 0 trials
- Bethlem myopathy 2 0 trials
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1 sub-type
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Oculopharyngodistal myopathy 0 trials
5 sub-types
- Oculopharyngodistal myopathy 1 0 trials
- Oculopharyngodistal myopathy 2 0 trials
- Oculopharyngodistal myopathy 3 0 trials
- Oculopharyngodistal myopathy 4 0 trials
- Oculopharyngodistal myopathy 5 0 trials
Most studied deeper sub-types
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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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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 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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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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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