Limb-girdle muscular dystrophy
MONDO:0016971Limb-girdle muscular dystrophy (LGMD) is a heterogeneous group of muscular dystrophies characterized by proximal weakness affecting the pelvic and shoulder girdles. Cardiac and respiratory impairment may be observed in certain forms of LGMD.
Also known as: LGMD, Leyden-Mobius muscular dystrophy, limb-girdle muscular dystrophy
31 clinical trials for this condition and its sub-types, 17 tagged with Limb-girdle muscular dystrophy itself.
Follow this condition to get notified about new trialsWhere it sits in the disease tree
Browse by category →Sub-types of Limb-girdle muscular dystrophy
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Autosomal recessive limb-girdle muscular dystrophy 0 trials · 18 incl. sub-types
32 sub-types
- Autosomal recessive limb-girdle muscular dystrophy type 2I 8 trials
- Autosomal recessive limb-girdle muscular dystrophy type 2A 5 trials
- Autosomal recessive limb-girdle muscular dystrophy type 2E 5 trials
- Autosomal recessive limb-girdle muscular dystrophy type 2C 4 trials
- Autosomal recessive limb-girdle muscular dystrophy type 2B 3 trials
- Autosomal recessive limb-girdle muscular dystrophy type 2D 3 trials
- Autosomal recessive limb-girdle muscular dystrophy type 2F 2 trials
- Autosomal recessive limb-girdle muscular dystrophy type 2L 2 trials
- Autosomal recessive limb-girdle muscular dystrophy type 2G 1 trial
- Autosomal recessive limb-girdle muscular dystrophy type 2J 1 trial
- Autosomal recessive limb-girdle muscular dystrophy type 2K 1 trial
- Autosomal recessive limb-girdle muscular dystrophy type 2M 1 trial
- Autosomal recessive limb-girdle muscular dystrophy type 2N 1 trial
- Autosomal recessive limb-girdle muscular dystrophy type 2O 1 trial
- Autosomal recessive limb-girdle muscular dystrophy type 2P 1 trial
- Autosomal recessive limb-girdle muscular dystrophy type 2Q 1 trial
- Autosomal recessive limb-girdle muscular dystrophy type 2R1 1 trial
- Autosomal recessive limb-girdle muscular dystrophy type 2T 1 trial
- Autosomal recessive limb-girdle muscular dystrophy type 2U 1 trial
- Autosomal recessive limb-girdle muscular dystrophy type 2W 1 trial
- Autosomal recessive limb-girdle muscular dystrophy type 2X 1 trial
- Autosomal recessive limb-girdle muscular dystrophy type 2Y 1 trial
- Autosomal recessive limb-girdle muscular dystrophy type R18 1 trial
- Autosomal recessive limb-girdle muscular dystrophy type 2H 0 trials
- Epidermolysis bullosa simplex 5B, with muscular dystrophy 0 trials
- Limb-girdle muscular dystrophy due to POMK deficiency 0 trials
- Muscular dystrophy, limb-girdle, autosomal recessive 23 0 trials
- Muscular dystrophy, limb-girdle, autosomal recessive 26 0 trials
- Muscular dystrophy, limb-girdle, autosomal recessive 27 0 trials
- Muscular dystrophy, limb-girdle, autosomal recessive 28 0 trials
- Muscular dystrophy, limb-girdle, autosomal recessive 29 0 trials
- Muscular dystrophy-dystroglycanopathy (limb-girdle), type C, 8 0 trials
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Muscular dystrophy, limb-girdle, autosomal dominant 0 trials · 4 incl. sub-types
8 sub-types
- Emery-Dreifuss muscular dystrophy 2, autosomal dominant 3 trials
- Autosomal dominant limb-girdle muscular dystrophy type 1D (DNAJB6) 2 trials
- Autosomal dominant limb-girdle muscular dystrophy type 1F 1 trial
- Autosomal dominant limb-girdle muscular dystrophy type 1G 1 trial
- Autosomal dominant limb-girdle muscular dystrophy type 1H 1 trial
- Autosomal dominant limb-girdle muscular dystrophy type 1E (DES) 0 trials
- Muscular dystrophy, limb-girdle, autosomal dominant 4 0 trials
- Myofibrillar myopathy 3 0 trials
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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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Experimental drug targets root cause of rare muscle-wasting disease
Disease control OngoingThis study tests an experimental drug, BBP-418, in people with limb girdle muscular dystrophy type 2I (LGMD2I), a rare genetic condition that causes progressive muscle weakness. The drug aims to fix a molecular defect by helping a faulty enzyme work better. The trial includes bot…
Phase 2 • Sponsor: ML Bio Solutions, Inc. • Aim: Disease control
Last updated Jul 23, 2026 00:00 UTC
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One-Time gene therapy aims to halt rare muscle disease
Disease control OngoingThis study tests a single dose of SRP-9003 gene therapy in 17 people with limb girdle muscular dystrophy 2E/R4, a genetic muscle-weakening disease. The goal is to restore a missing protein in muscle cells and improve muscle function. Both walkers and non-walkers can join, and the…
Phase 3 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated Jun 27, 2026 09:10 UTC
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Experimental gene therapy targets rare muscle disease in first human test
Disease control OngoingThis early-phase trial tests a single-dose gene therapy called SRP-9003 in 6 people with limb girdle muscular dystrophy type 2E/R4, a rare genetic muscle-weakening disease. The main goals are to check safety and see if the therapy can produce the missing beta-sarcoglycan protein …
Phase 1 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated Jun 27, 2026 09:06 UTC
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Can MRI scans reveal how ANO5 muscle disease progresses?
Knowledge-focused OngoingThis study follows adults with pathogenic variants in the anoctamin 5 gene, which causes a form of limb-girdle muscular dystrophy. Over three years, researchers will use whole-body MRI to measure changes in muscle fat content, and questionnaires to track fatigue and quality of li…
Sponsor: Rigshospitalet, Denmark • Aim: Knowledge-focused
Last updated Aug 14, 2026 00:00 UTC
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Can MRI scans reveal the hidden pattern of muscle damage in a rare muscular dystrophy?
Knowledge-focused OngoingThis study aims to describe the pattern of muscle involvement in people with limb girdle muscular dystrophy caused by mutations in the anoctamin 5 gene. Researchers will collect and analyze MRI scans from 200 participants to see which muscles are affected and how much fat replace…
Sponsor: Rigshospitalet, Denmark • Aim: Knowledge-focused
Last updated Aug 14, 2026 00:00 UTC
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Paving the way: new study aims to sharpen tools for LGMD R1 trials
Knowledge-focused OngoingThis 24-month observational study follows 100 people aged 12–50 with Limb Girdle Muscular Dystrophy type R1 (LGMD R1). Researchers will test whether a motor function scale called NSAD and muscle fat measurements from MRI can reliably track disease progression. The goal is to vali…
Sponsor: Virginia Commonwealth University • Aim: Knowledge-focused
Last updated Jun 27, 2026 13:07 UTC
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New study tracks muscle decline in rare muscular dystrophy
Knowledge-focused OngoingThis study follows 25 people with limb-girdle muscular dystrophy type 2A (LGMD2A), a rare genetic disease that causes progressive muscle weakness. Researchers will measure how muscle strength changes over time and how it affects quality of life. The goal is to better understand t…
Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:11 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