Disease · EFO_0003867
rhabdomyolysis
6 sourcesdiseases-2026-09-18-0.1.0
Cite this record
ConductScience Library. rhabdomyolysis. Diseases, record EFO_0003867. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/EFO_0003867
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.
Drugs7
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 |
|---|---|---|---|---|---|---|---|---|
| DANTROLENE | CHEMBL1201288 | Small molecule | Phase 2/3 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| TRIHEPTANOIN | CHEMBL4297585 | Small molecule | Phase 2 | ClinicalTrials.gov | 2 | 2 | 2 | Drugs and targets |
| DEXTROSE | CHEMBL1222250 | Small molecule | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| POTASSIUM | CHEMBL1201290 | Small molecule | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| DEXAMETHASONE | CHEMBL384467 | Small molecule | Phase 1 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| HYDROXYCHLOROQUINE SULFATE | CHEMBL1690 | Small molecule | Unknown | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| SIMVASTATIN | CHEMBL1064 | Small molecule | Unknown | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
Targets6
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 |
|---|---|---|---|---|---|---|
| HMGCR | 3-hydroxy-3-methylglutaryl-coenzyme A reductase | 1 | HMG-CoA reductase inhibitor | Unknown | https://conductscience.org/library/genes/3156 | Drugs and targets |
| NR3C1 | Glucocorticoid receptor | 1 | Glucocorticoid receptor agonist | Phase 1 | https://conductscience.org/library/genes/2908 | Drugs and targets |
| RYR1 | Ryanodine receptor 1 | 1 | Ryanodine receptor 1 antagonist | Phase 2/3 | https://conductscience.org/library/genes/6261 | Drugs and targets |
| RYR3 | Ryanodine receptor 3 | 1 | Ryanodine receptor 3 antagonist | Phase 2/3 | https://conductscience.org/library/genes/6263 | Drugs and targets |
| TLR7 | Toll-like receptor 7 | 1 | Toll-like receptor 7 antagonist | Unknown | https://conductscience.org/library/genes/51284 | Drugs and targets |
| TLR9 | Toll-like receptor 9 | 1 | Toll-like receptor 9 antagonist | Unknown | https://conductscience.org/library/genes/54106 | Drugs and targets |
Trials7
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 |
|---|---|---|---|---|---|---|---|---|
| NCT02214160 | Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Extension Study for Subjects Previously Enrolled in Triheptanoin Studies | Phase 2 | 94 | 2021-12-08 | Carnitine Palmitoyltransferase (CPT I or CPT II) Deficiency, Very Long Chain Acyl-CoA Dehydrogenase (VLCAD) Deficiency, Long-chain 3-hydroxy-acyl-CoA Dehydrogenase (LCHAD) Deficiency, Trifunctional Protein (TFP) Deficiency, Carnitine-acylcarnitine Translocase (CACT) Deficiency | TRIHEPTANOIN | Its record names the disease | ClinicalTrials.gov |
| NCT01379625 | Study of Triheptanoin for Treatment of Long-Chain Fatty Acid Oxidation Disorder | Phase 2 | 32 | 2017-03-23 | Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency, Carnitine Palmitoyltransferase 2 (CPT2) Deficiency, Mitochondrial Trifunctional Protein (TFP) Deficiency, Long-chain 3 hydroxyacylCoA Dehydrogenase (LCHAD) Deficiency | TRIHEPTANOIN | Its record names the disease | ClinicalTrials.gov |
| NCT06429982 | Dexamethasone Use in Pediatric Rhabdomyolysis Patients in Addition to Standard Protocols | Phase 1 | 50 | Rhabdomyolysis | DEXAMETHASONE | Its record names the disease | ClinicalTrials.gov | |
| NCT06966843 | Dantrolene in Statin-induced Myopathy | Phase 2/3 | 23 | Statins Induced Myopathy | DANTROLENE | Its record names the disease | ClinicalTrials.gov | |
| NCT00990834 | Muscle Characteristics Associated With Statin Therapy | Not applicable | 0 | Hydroxymethylglutaryl-CoA Reductase Inhibitors, Myopathy | SIMVASTATIN | Its record names the disease | ClinicalTrials.gov | |
| NCT04007562 | Acute Rhabdomyolysis and Muscle Pain Associated With Mutations in the LPIN1 Gene - A Retrospective Study Describing the Safety and Efficacy of Hydroxychloroquine Sulfate Given on a Compassionate Basis to Patients Suffering From Lipin-1 Deficiency | 0 | LIPIN1 Deficiency | HYDROXYCHLOROQUINE SULFATE | Its record names the disease | ClinicalTrials.gov | ||
| NCT05627479 | MAGIK for Femoral/Tibial Shaft Fractures | Phase 2 | 0 | Femoral Shaft Fracture, Tibial Shaft Fracture | DEXTROSE, POTASSIUM | Its record names the disease | ClinicalTrials.gov |
Comparisons in its trials1
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 |
|---|---|---|---|---|
| Medium Chain Triglyceride (MCT) vs Triheptanoin: serious adverse events | 0 of 16 | 0 of 16 | 32 | NCT01379625 |
Source Trial comparisons
Papers from its trials2
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/EFO_0003867
GET https://conductscience.org/api/diseases/v1/EFO_0003867/drugs