Disease · MONDO_0016971

limb-girdle muscular dystrophy

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. limb-girdle muscular dystrophy. Diseases, record MONDO_0016971. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0016971

On this page

Disease

Names and IDs as Open Targets gives them. Its IDs in MeSH, Orphanet, the Disease Ontology and the NCI Thesaurus are those Open Targets states for it.

Name: limb-girdle muscular dystrophy; Open Targets
Open Targets ID: MONDO_0016971; Open Targets
Other names: Erb's muscular dystrophy, LGMD, Leyden-Mbius muscular dystrophy, Leyden-Mobius muscular dystrophy, Limb Girdle Muscular Dystrophies, Limb-girdle muscular dystrophy (disorder), Muscular Dystrophies, Limb Girdle, Muscular Dystrophy, Limb Girdle, calpainopathy; Open Targets
Disease Ontology: DOID:11724; Disease Ontology
MeSH: D049288; MeSH
NCI Thesaurus: C84828; NCI Thesaurus
Orphanet: ORPHA:263; Orphanet
Highest stage of its drugs: Phase 3, for 1 of its 4 drugs, from TTD; Open Targets

Drugs4

The drugs Open Targets' clinical reports study for the disease, the highest stage first, then the most trial reports. The stage is Open Targets', the highest any of its sources reports for the pair, and each row names the sources that report it.

Drug	ChEMBL ID	Type	Open Targets' stage	Stage reported by	Reports	Of them trials	Trials shown here	Source
RIBITOL	CHEMBL3137744	Small molecule	Phase 3	TTD	2	0	0	Drugs and targets
PREDNISONE	CHEMBL635	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
ATYR-1940	CHEMBL4297550		Phase 1/2	ClinicalTrials.gov	2	2	2	Drugs and targets
BIDRIDISTROGENE XEBOPARVOVEC	CHEMBL4650240	Gene	Phase 1/2	TTD	2	0	0	Drugs and targets

Targets1

The targets ChEMBL's mechanism records give for the disease's drugs, through Open Targets, the most drugs first. A mechanism record says what a drug acts on, not that the target causes the disease.

Target	Protein	Its drugs that act on it	Mechanisms	Highest stage of those drugs	Gene in Genes
NR3C1	Glucocorticoid receptor	1	Glucocorticoid receptor agonist	Phase 2	https://conductscience.org/library/genes/2908

Source Drugs and targets

Trials3

Trials an Open Targets clinical report links to the disease, posted results first, then the largest. A trial is shown when the release's checks confirm its link to the disease and Drugs and targets shows its link to one of the disease's drugs.

Trial	Title	Phase	Participants	Results first posted	Conditions	Drugs	Linked by	Source
NCT04054375	Weekly Steroids in Muscular Dystrophy	Phase 2	20	2021-07-08	Limb-girdle Muscular Dystrophy, Becker Muscular Dystrophy	PREDNISONE	Its record names the disease	ClinicalTrials.gov
NCT02579239	Evaluate Safety and Biological Activity of ATYR1940 in Participants With Limb Girdle Muscular Dystrophy 2B (LGMD2B) and Facioscapulohumeral Muscular Dystrophy (FSHD)	Phase 1/2	18	2023-10-19	Limb-Girdle Muscular Dystrophies, Facioscapulohumeral Muscular Dystrophy	ATYR-1940	Its record names the disease	ClinicalTrials.gov
NCT02836418	Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Participants With Limb Girdle and Facioscapulohumeral Muscular Dystrophy (FSHD)	Phase 1/2	8	2023-12-22	Facioscapulohumeral Muscular Dystrophy, Limb Girdle Muscular Dystrophy	ATYR-1940	Its record names the disease	ClinicalTrials.gov

Comparisons in its trials5

Comparisons Trial comparisons calculated in the trials shown here, most participants first. A comparison in one of these trials does not always compare a treatment for the disease, so read the two groups.

Comparison	Group 1, with the outcome	Group 2, with the outcome	Participants	Trial	Source
Group A - FSHD vs Group B - LGMD2B and FSHD: Number of Participants With Vital Sign Abnormality Resulting in a TEAE	0 of 4	3 of 14	18	NCT02579239	Trial comparisons
Group A - FSHD vs Group B - LGMD2B and FSHD: Number of Participants With a Clinical Laboratory Abnormality Leading to an AE	0 of 4	4 of 14	18	NCT02579239	Trial comparisons
Group A - FSHD vs Group B - LGMD2B and FSHD: Number of Participants With a Clinically Significant Pulmonary Function Event Resulting in a TEAE	0 of 4	0 of 14	18	NCT02579239	Trial comparisons
Group A - FSHD vs Group B - LGMD2B and FSHD: Number of Participants With a Clinically Significant Electrocardiogram (ECG) Abnormality Leading to a TEAE	0 of 4	0 of 14	18	NCT02579239	Trial comparisons
Group A - FSHD vs Group B - LGMD2B and FSHD: serious adverse events	0 of 4	0 of 14	18	NCT02579239	Trial comparisons

Papers from its trials0

The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.

None in this release.

Data dates

The date of each source this release read.

Open Targets: 2026-09-18; Open Targets
Drug links and their checks, from Drugs and targets: 2026-09-18; Drugs and targets
ClinicalTrials.gov, from Trials: 2026-11-12; Trials
Comparisons, from Trial comparisons: 2026-03-23; Trial comparisons
PubMed, for the checks: 2026-11-06; PubMed
MeSH, for the checks: 2026-11-10; MeSH

Sources 6

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ChEMBL, through Open TargetsCC BY-SA 3.0
ClinicalTrials.govNo license stated
NLM's MeSHNLM's terms
Open TargetsCC0 1.0
PubMedNLM's terms

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ChEMBL, through Open TargetsCC BY-SA 3.0, for the parts from ChEMBL. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
ClinicalTrials.govnone stated
NLM's MeSHNLM's
Open TargetsCC0 1.0. Open Targets asks that users cite its latest paper: Buniello et al. 2025, Open Targets Platform: facilitating therapeutic hypotheses building in drug discovery, Nucleic Acids Research 53(D1):D1467, PMID 39657122. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
PubMedNLM's terms

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this disease from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/diseases/v1/MONDO_0016971

GET https://conductscience.org/api/diseases/v1/MONDO_0016971/drugs

API reference For AI assistants