Disease · MONDO_0001347
facioscapulohumeral muscular dystrophy
6 sourcesdiseases-2026-09-18-0.1.0
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ConductScience Library. facioscapulohumeral muscular dystrophy. Diseases, record MONDO_0001347. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0001347
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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.
- Other names
Atrophies, Facioscapulohumeral, Atrophy, Facioscapulohumeral, Dystrophies, Facioscapulohumeral Muscular, Dystrophies, Landouzy-Dejerine, Dystrophy, Facioscapulohumeral Muscular, Dystrophy, Landouzy-Dejerine, FMD - Facioscapulohumeral muscular dystrophy, FSH - Facioscapulohumeral muscular dystrophy, FSH dystrophy, FSHD, FSHD - Facioscapulohumeral muscular dystrophy, Facioscapulohumeral Atrophies, Facioscapulohumeral Atrophy, Facioscapulohumeral Muscular Dystrophies, Facioscapulohumeral Type Progressive Muscular Dystrophy, Facioscapulohumeral muscular dystrophy (disorder), Fascioscapulohumeral muscular dystrophy, Landouzy Dejerine Dystrophy, Landouzy Dejerine muscular dystrophy, Landouzy-Dejerine Dystrophies, Landouzy-Dejerine myopathy, Muscular Dystrophies, Facioscapulohumeral, Muscular Dystrophy, Facioscapulohumeral, Muscular dystrophy, Landouzy-Dejerine, Progressive Muscular Dystrophy, Facioscapulohumeral Type, facioscapulohumeral dystrophy, facioscapulohumeral myopathy
- Open Targets
Drugs11
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 |
|---|---|---|---|---|---|---|---|---|
| LOSMAPIMOD | CHEMBL1088752 | Small molecule | Phase 3 | ClinicalTrials.gov | 4 | 4 | 4 | Drugs and targets |
| EFMITERMANT ALFA | CHEMBL4297867 | Protein | Phase 2 | ClinicalTrials.gov | 2 | 2 | 2 | Drugs and targets |
| EMUGROBART | CHEMBL6068245 | Antibody | Phase 2 | ClinicalTrials.gov and TTD | 2 | 1 | 1 | Drugs and targets |
| APITEGROMAB | CHEMBL4594326 | Antibody | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| SATRALIZUMAB | CHEMBL3833307 | Antibody | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| ATYR-1940 | CHEMBL4297550 | Phase 1/2 | ClinicalTrials.gov | 4 | 4 | 4 | Drugs and targets | |
| CLENBUTEROL | CHEMBL49080 | Small molecule | Phase 1 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| SOMATROPIN | CHEMBL1201621 | Protein | Phase 1 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| TESTOSTERONE | CHEMBL386630 | Small molecule | Phase 1 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| ALBUTEROL | CHEMBL714 | Small molecule | Unknown | ClinicalTrials.gov | 2 | 2 | 2 | Drugs and targets |
1 to 10 of 11
See all 11 drugsTargets8
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 | Source |
|---|---|---|---|---|---|---|
| ADRB2 | Beta-2 adrenergic receptor | 2 | Beta-2 adrenergic receptor agonist | Phase 1 | https://conductscience.org/library/genes/154 | Drugs and targets |
| AR | Androgen receptor | 2 | Androgen Receptor agonist | Phase 1 | https://conductscience.org/library/genes/367 | Drugs and targets |
| GHR | Growth hormone receptor | 1 | Growth hormone receptor agonist | Phase 1 | https://conductscience.org/library/genes/2690 | Drugs and targets |
| IL6R | IL6Ralpha/GP130 | 1 | IL6Ralpha/GP130 antagonist | Phase 2 | https://conductscience.org/library/genes/3570 | Drugs and targets |
| IL6ST | IL6Ralpha/GP130 | 1 | IL6Ralpha/GP130 antagonist | Phase 2 | https://conductscience.org/library/genes/3572 | Drugs and targets |
| MAPK11 | Mitogen-activated protein kinase 11 | 1 | MAP kinase p38 beta inhibitor | Phase 3 | https://conductscience.org/library/genes/5600 | Drugs and targets |
| MAPK14 | Mitogen-activated protein kinase 14 | 1 | MAP kinase p38 alpha inhibitor | Phase 3 | https://conductscience.org/library/genes/1432 | Drugs and targets |
| MSTN | Growth/differentiation factor 8 | 1 | Growth/differentiation factor 8 inhibitor | Phase 2 | https://conductscience.org/library/genes/2660 | Drugs and targets |
Trials17
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 |
|---|---|---|---|---|---|---|---|---|
| NCT05397470 | Efficacy and Safety of Losmapimod in Treating Participants With Facioscapulohumeral Muscular Dystrophy (FSHD) (REACH) | Phase 3 | 260 | 2025-11-10 | Facioscapulohumeral Muscular Dystrophy (FSHD) | LOSMAPIMOD | Its record names the disease | ClinicalTrials.gov |
| NCT02927080 | Study of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) | Phase 2 | 95 | 2021-04-22 | Facioscapulohumeral Muscular Dystrophy | EFMITERMANT ALFA | Its record names the disease | ClinicalTrials.gov |
| NCT04003974 | Efficacy and Safety of Losmapimod in Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD) | Phase 2 | 80 | 2024-07-10 | Facioscapulohumeral Muscular Dystrophy (FSHD) | LOSMAPIMOD | Its record names the disease | ClinicalTrials.gov |
| NCT04264442 | Efficacy and Safety of Losmapimod in Treating Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD) With Open-Label Extension (OLE) | Phase 2 | 76 | 2025-09-30 | Facioscapulohumeral Muscular Dystrophy (FSHD) | LOSMAPIMOD | Its record names the disease | ClinicalTrials.gov |
| NCT03943290 | Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) and Charcot-Marie Tooth (CMT) Disease Types 1 and X (CMT1 and CMTX) | Phase 2 | 62 | 2021-06-02 | Facioscapulohumeral Muscular Dystrophy, Charcot-Marie-Tooth Disease | EFMITERMANT ALFA | Its record names the disease | ClinicalTrials.gov |
| NCT02239224 | Safety, Tolerability, Pharmacokinetics, and Biological Activity of ATYR1940 in Adult Participants With Muscular Dystrophy | Phase 1/2 | 20 | 2021-08-11 | Facioscapulohumeral Muscular Dystrophy (FSHD) | ATYR-1940 | Its record names the disease | ClinicalTrials.gov |
| NCT03123913 | Study of Testosterone and rHGH in FSHD | Phase 1 | 20 | 2023-05-16 | Facioscapulohumeral Muscular Dystrophy | SOMATROPIN, TESTOSTERONE | 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 |
| NCT04004000 | Evaluation of Safety, Tolerability, and Changes in Biomarker and Clinical Outcome Assessments of Losmapimod for FSHD1 With Extension | Phase 2 | 14 | 2025-12-02 | Facioscapulohumeral Muscular Dystrophy 1 | LOSMAPIMOD | Open Targets' reading of its record | ClinicalTrials.gov |
| NCT02603562 | Evaluate Safety and Biological Activity of ATYR1940 in Participants With Early Onset Facioscapulohumeral Muscular Dystrophy | Phase 1/2 | 8 | 2023-10-19 | Facioscapulohumeral Muscular Dystrophy (FSHD) | ATYR-1940 | Its record names the disease | ClinicalTrials.gov |
1 to 10 of 17
See all 17 trialsComparisons in its trials15
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 |
|---|---|---|---|---|---|
| Part A: Losmapimod 15 Milligrams (mg) vs Part A: Placebo: Part A: Number of Participants With Clinically Significant Changes in Vital Parameters | 0 of 130 | 0 of 130 | 260 | NCT05397470 | Trial comparisons |
| Part A: Losmapimod 15 Milligrams (mg) vs Part A: Placebo: Part A: Number of Participants With Clinically Significant Changes in Physical Examinations | 0 of 130 | 0 of 130 | 260 | NCT05397470 | Trial comparisons |
| Part A: Losmapimod 15 Milligrams (mg) vs Part A: Placebo: Part A: Number of Participants With Clinically Significant Changes in Electrocardiogram (ECG) Parameters | 0 of 130 | 0 of 130 | 260 | NCT05397470 | Trial comparisons |
| Part A: Losmapimod 15 Milligrams (mg) vs Part A: Placebo: Part A: Number of Participants With Clinically Significant Changes in Urinalysis | 0 of 130 | 0 of 130 | 260 | NCT05397470 | Trial comparisons |
| Part A: Losmapimod 15 Milligrams (mg) vs Part A: Placebo: Part A: Number of Participants With Clinically Significant Changes in Hematology Parameters | 0 of 130 | 0 of 130 | 260 | NCT05397470 | Trial comparisons |
| Part A: Losmapimod 15 Milligrams (mg) vs Part A: Placebo: Part A: Number of Participants With Clinically Significant Changes in Clinical Chemistry Parameters | 0 of 130 | 0 of 130 | 260 | NCT05397470 | Trial comparisons |
| Losmapimod 15 Milligrams (mg) Twice Daily (BID) vs Placebo: Number of Participants With Adverse Events of Special Interest (AESIs) | 0 of 40 | 0 of 40 | 80 | NCT04003974 | Trial comparisons |
| Losmapimod 15 Milligrams (mg) Twice Daily (BID) vs Placebo: deaths from any cause | 0 of 40 | 0 of 40 | 80 | NCT04003974 | Trial comparisons |
| Losmapimod 15 Milligrams (mg) Twice Daily (BID) vs Placebo: Number of Participants Who Prematurely Discontinued Study Drug Due to a Treatment Emergent Adverse Event (TEAE) | 0 of 40 | 0 of 40 | 80 | NCT04003974 | Trial comparisons |
| Losmapimod 15 Milligrams (mg) Twice Daily (BID) vs Placebo: serious adverse events | 2 of 40 | 0 of 40 | 80 | NCT04003974 | Trial comparisons |
1 to 10 of 15
See all 15 comparisons in its trialsPapers from its trials4
The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.
Data dates
The date of each source this release read.
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_0001347
GET https://conductscience.org/api/diseases/v1/MONDO_0001347/drugs