Condition (MeSH)
Muscular Dystrophies, Limb-Girdle
ClinicalTrials.gov lists 49 trials for Muscular Dystrophies, Limb-Girdle or a narrower condition beneath it. Sarepta Therapeutics, Inc. has the most among industry and government lead sponsors, with 9.
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Trials per phase (log scale)
46 of the 49 trials are tagged with Muscular Dystrophies, Limb-Girdle itself; the rest are tagged with a narrower condition beneath it.
When did Muscular Dystrophies, Limb-Girdle trials start? (17)
Shown in this order: most recent first, 1990 onwards. 2026 is partial: trials registered with a 2026 start date so far, not a full year.
Trials over time, by phase
- Phase 1
- Phase 1/2
- Phase 2
- Phase 2/3
- Phase 3
- Not applicable
- Observational
Top of scale: 5
Trials registered with a start date in each year, 1990 onward, stacked by phase (complete years only; the current partial year is in the table's notes). The table below lists the same counts, most recent years first.
| Year | Trials started |
|---|---|
| 2026 (partial year) | 1 |
| 2025 | 3 |
| 2024 | 5 |
| 2023 | 4 |
| 2022 | 3 |
| 2021 | 5 |
| 2020 | 1 |
| 2019 | 4 |
| 2018 | 3 |
| 2016 | 4 |
Showing 10 of 17 earlier years.
No forecast is made for this condition: it averages 3.2 trial starts a year over the five years to September, under the 5 a year the method needs.
Recruitment duration, a model prediction: 3 trials still running at the 2026-10-09 snapshot, fewer than the 5 needed for a median. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Muscular Dystrophies, Limb-Girdle trials in? (7)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 8 |
| Phase 1/2 | 6 |
| Phase 2 | 2 |
| Phase 2/3 | 1 |
| Phase 3 | 3 |
| Not applicable | 5 |
| Observational | 24 |
How many Muscular Dystrophies, Limb-Girdle trials are recruiting? (7)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Recruiting | 4 |
| Enrolling by invitation | 1 |
| Active, not recruiting | 9 |
| Completed | 19 |
| Terminated | 5 |
Showing 5 of 7 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Muscular Dystrophies, Limb-Girdle trials? (7)
Shown in this order: most trials first. Lead sponsors the registry classes as industry, NIH, US federal, other government or network. A hospital or university the registry files as government can appear here. Sponsors it classes as academic or other are shown on the full page, because that class cannot separate an institution from a named investigator.
| Sponsor | Trials |
|---|---|
| Sarepta Therapeutics, Inc. | 9 |
| Cooperative International Neuromuscular Research Group | 4 |
| ML Bio Solutions, Inc. | 2 |
| aTyr Pharma, Inc. | 2 |
| AbiliTech Medical Inc. | 1 |
Showing 5 of 7 sponsors.
Which drugs are being tested for Muscular Dystrophies, Limb-Girdle? (8)
Shown in this order: most trials first. One row per drug, matched to a single molecule under all its names, ranked by number of trials.
| Drug | Trials |
|---|---|
| SRP-9003 | 3 |
| SRP-9004 | 2 |
| ATYR1940 | 2 |
| BBP-418 | 1 |
| Deflazacort | 1 |
Showing 5 of 8 drugs.
Interventions as named in the registry (7)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| Glucocorticoids | 1 |
| Autologous bone marrow mononuclear cell transplantation | 1 |
| Adjuvant Chemotherapy | 1 |
| Coenzyme Q10 and Lisinopril | 1 |
| Stem Cell | 1 |
Showing 5 of 7 interventions.
These are the names sponsors entered on ClinicalTrials.gov that we have not matched to a single molecule. Some are drugs under a brand or code name; others are procedures, doses or arm labels.
Which serious adverse events do Muscular Dystrophies, Limb-Girdle trials report? (3)
Shown in this order: most subjects affected first. Subjects affected summed across trials, a cross-trial signal, not a rate.
| Adverse event | Subjects affected | Trials |
|---|---|---|
| Rhabdomyolysis | 2 | 1 |
| Femur fracture | 1 | 1 |
| Myoglobinuria | 1 | 1 |
Which publications cover Muscular Dystrophies, Limb-Girdle? (72)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Recurrent <i>CAPN3</i> p.Asp753Asn Variant Supports a Potential Dominant Calpainopathy with Variable Clinical Expressivity. | International journal of molecular sciences | 2025 | Yes |
| Gene therapy with bidridistrogene xeboparvovec for limb-girdle muscular dystrophy type 2E/R4: phase 1/2 trial results. | Nature medicine | 2024 | Yes |
| Miyoshi myopathy associated with spine rigidity and multiple contractures: a case report. | BMC musculoskeletal disorders | 2024 | Yes |
| Defining clinical endpoints in limb girdle muscular dystrophy: a GRASP-LGMD study. | BMC neurology | 2024 | Yes |
| Asymptomatic and oligosymptomatic states of dysferlinopathy. | Journal of neuromuscular diseases | 2024 | Yes |
Showing 5 of 72 publications.
Prevalence (24)
Shown in this order: jurisdiction, then disease name. Orphanet observation for the mapped ORPHA concept; the mapping relation column says whether that concept is exactly this condition or broader or narrower.
| Orphanet disease | Type | Class | Geography | Jurisdiction | Mapping relation |
|---|---|---|---|---|---|
| Limb-girdle muscular dystrophy | Point prevalence | 1-9 / 100 000 | United Kingdom | GB | Exact |
| Limb-girdle muscular dystrophy | Point prevalence | 1-9 / 100 000 | United Kingdom | GB | Exact |
| Limb-girdle muscular dystrophy | Point prevalence | 1-9 / 100 000 | United Kingdom | GB | Unspecified |
| Limb-girdle muscular dystrophy | Point prevalence | 1-9 / 100 000 | Ireland | Not stated | Exact |
| Limb-girdle muscular dystrophy | Point prevalence | 1-9 / 1 000 000 | Netherlands | Not stated | Exact |
Showing 5 of 24 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Limb-girdle muscular dystrophy | 263 | Exact | Orphadata Science knowledge files, Product 1 |
| Limb-girdle muscular dystrophy | 263 | Exact | Orphanet Rare Disease Ontology (ORDO) |
Sub-conditions in MeSH (1)
MeSH descendants one level down with at least one trial, most trials first.
Not on this page
Not measured: delivery record.