Disease · MONDO_0004976
amyotrophic lateral sclerosis
6 sourcesdiseases-2026-09-18-0.1.0
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ConductScience Library. amyotrophic lateral sclerosis. Diseases, record MONDO_0004976. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0004976
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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
ALS, ALS (Amyotrophic Lateral Sclerosis), AMYOTROPHIC SCLEROSIS, Amyotrophic Lateral Sclerosis With Dementia, Amyotrophic Lateral Sclerosis, Guam Form, Amyotrophic Lateral Sclerosis-Parkinsonism/dementia Complex 1, Amyotrophic lateral sclerosis (disorder), Amyotrophic lateral sclerosis, Parkinsonism/Dementia complex of Guam, Bulbar motor neuron disease, Charcot disease, Dementia With Amyotrophic Lateral Sclerosis, Disease, Lou-Gehrigs, GEHRIGS DIS, Gehrig Disease, Gehrig's Disease, Gehrigs Disease, Guam Form of Amyotrophic Lateral Sclerosis, LOU GEHRIG DIS, LOU GEHRIGS DIS, Lateral Scleroses, Amyotrophic, Lou Gehrig disease, Lou Gehrig's Disease, Lou Gehrigs Disease, MOTOR NEURON DIS AMYOTROPHIC LATERAL SCLEROSIS, Motor Neuron Disease, Amyotrophic Lateral Sclerosis, Motor neuron disease, bulbar, Sclerosis, Amyotrophic Lateral
- Open Targets
- Highest stage of its drugs
- Approval, for 5 of its 185 drugs, from ClinicalTrials.gov, DailyMed, EMA Human Drugs, FDA, PMDA and TTD
- Open Targets
Drugs185
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 |
|---|---|---|---|---|---|---|---|---|
| RILUZOLE | CHEMBL744 | Small molecule | Approval | DailyMed, EMA Human Drugs and TTD | 32 | 23 | 23 | Drugs and targets |
| EDARAVONE | CHEMBL290916 | Small molecule | Approval | DailyMed, FDA, PMDA and TTD | 18 | 13 | 13 | Drugs and targets |
| SODIUM PHENYLBUTYRATE | CHEMBL1746 | Small molecule | Approval | ClinicalTrials.gov | 10 | 8 | 3 | Drugs and targets |
| TOFERSEN | CHEMBL3833346 | Oligonucleotide | Approval | ClinicalTrials.gov, EMA Human Drugs, FDA, PMDA and TTD | 10 | 6 | 6 | Drugs and targets |
| MECOBALAMIN | CHEMBL2359370 | Small molecule | Approval | PMDA | 4 | 2 | 2 | Drugs and targets |
| TIRASEMTIV | CHEMBL3039529 | Small molecule | Phase 3 | ClinicalTrials.gov and TTD | 7 | 6 | 6 | Drugs and targets |
| CREATINE | CHEMBL283800 | Small molecule | Phase 3 | ClinicalTrials.gov | 6 | 6 | 5 | Drugs and targets |
| ARIMOCLOMOL | CHEMBL2107726 | Small molecule | Phase 3 | ClinicalTrials.gov | 5 | 5 | 5 | Drugs and targets |
| LITHIUM CARBONATE | CHEMBL1200826 | Small molecule | Phase 3 | ClinicalTrials.gov | 5 | 5 | 4 | Drugs and targets |
| DEXPRAMIPEXOLE | CHEMBL249420 | Small molecule | Phase 3 | ClinicalTrials.gov | 4 | 4 | 4 | Drugs and targets |
1 to 10 of 185
See all 185 drugsTargets262
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 |
|---|---|---|---|---|---|---|
| SCN4A | Sodium channel alpha subunit | 6 | Sodium channel alpha subunit blocker; Sodium channel protein type IV alpha subunit blocker | Approval | https://conductscience.org/library/genes/6329 | Drugs and targets |
| SCN5A | Sodium channel alpha subunit | 6 | Sodium channel alpha subunit blocker; Sodium channel protein type V alpha subunit blocker | Approval | https://conductscience.org/library/genes/6331 | Drugs and targets |
| GRIN2A | Glutamate [NMDA] receptor | 5 | Glutamate [NMDA] receptor antagonist; Glutamate [NMDA] receptor negative allosteric modulator; Glutamate [NMDA] receptor subunit epsilon 1 antagonist | Phase 3 | https://conductscience.org/library/genes/2903 | Drugs and targets |
| SCN10A | Sodium channel alpha subunit | 5 | Sodium channel alpha subunit blocker | Approval | https://conductscience.org/library/genes/6336 | Drugs and targets |
| SCN11A | Sodium channel alpha subunit | 5 | Sodium channel alpha subunit blocker | Approval | https://conductscience.org/library/genes/11280 | Drugs and targets |
| SCN1A | Sodium channel alpha subunit | 5 | Sodium channel alpha subunit blocker | Approval | https://conductscience.org/library/genes/6323 | Drugs and targets |
| SCN2A | Sodium channel alpha subunit | 5 | Sodium channel alpha subunit blocker | Approval | https://conductscience.org/library/genes/6326 | Drugs and targets |
| SCN3A | Sodium channel alpha subunit | 5 | Sodium channel alpha subunit blocker | Approval | https://conductscience.org/library/genes/6328 | Drugs and targets |
| SCN7A | Sodium channel alpha subunit | 5 | Sodium channel alpha subunit blocker | Approval | https://conductscience.org/library/genes/6332 | Drugs and targets |
| SCN8A | Sodium channel alpha subunit | 5 | Sodium channel alpha subunit blocker | Approval | https://conductscience.org/library/genes/6334 | Drugs and targets |
1 to 10 of 262
See all 262 targetsTrials240
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. 32 more are not shown, since the checks do not confirm them.
| Trial | Title | Phase | Participants | Results first posted | Conditions | Drugs | Linked by | Source |
|---|---|---|---|---|---|---|---|---|
| NCT01281189 | Phase 3 Study of Dexpramipexole in ALS | Phase 3 | 942 | 2021-06-07 | Amyotrophic Lateral Sclerosis | DEXPRAMIPEXOLE | Its record names the disease | ClinicalTrials.gov |
| NCT02496767 | Ventilatory Investigation of Tirasemtiv and Assessment of Longitudinal Indices After Treatment for a Year | Phase 3 | 744 | 2020-06-19 | Amyotrophic Lateral Sclerosis | RILUZOLE, TIRASEMTIV | Its record names the disease | ClinicalTrials.gov |
| NCT01709149 | Study of Safety, Tolerability & Efficacy of CK-2017357 in Amyotrophic Lateral Sclerosis (ALS) | Phase 2 | 711 | 2020-03-31 | Amyotrophic Lateral Sclerosis | TIRASEMTIV | Its record names the disease | ClinicalTrials.gov |
| NCT01622088 | Phase 3 Extension Study of Dexpramipexole in ALS | Phase 3 | 616 | 2022-04-07 | Amyotrophic Lateral Sclerosis | DEXPRAMIPEXOLE | Its record names the disease | ClinicalTrials.gov |
| NCT00349622 | Clinical Trial Ceftriaxone in Subjects With ALS | Phase 3 | 513 | 2014-04-01 | Amyotrophic Lateral Sclerosis, ALS | CEFTRIAXONE | Its record names the disease | ClinicalTrials.gov |
| NCT00868166 | Safety and Efficacy of TRO19622 as add-on Therapy to Riluzole Versus Placebo in Treatment of Patients Suffering From ALS | Phase 3 | 512 | 2019-02-15 | Amyotrophic Lateral Sclerosis | OLESOXIME, RILUZOLE | Its record names the disease | ClinicalTrials.gov |
| NCT03505021 | Effects of Oral Levosimendan (ODM-109) on Respiratory Function in Patients With ALS | Phase 3 | 496 | 2021-08-31 | Amyotrophic Lateral Sclerosis | LEVOSIMENDAN | Its record names the disease | ClinicalTrials.gov |
| NCT04944784 | A Study to Evaluate the Efficacy and Safety of Reldesemtiv in Patients With Amyotrophic Lateral Sclerosis (ALS) | Phase 3 | 489 | 2024-12-05 | Amyotrophic Lateral Sclerosis | RELDESEMTIV | Its record names the disease | ClinicalTrials.gov |
| NCT03160898 | A Study to Evaluate Efficacy, Safety and Tolerability of CK-2127107 in Patients With Amyotrophic Lateral Sclerosis (ALS) | Phase 2 | 458 | 2020-09-11 | Amyotrophic Lateral Sclerosis | RELDESEMTIV | Its record names the disease | ClinicalTrials.gov |
| NCT04569084 | Efficacy and Safety Study of Oral Edaravone Administered in Subjects With ALS | Phase 3 | 384 | 2025-03-27 | ALS | EDARAVONE | Its record names the disease | ClinicalTrials.gov |
1 to 10 of 240
See all 240 trialsComparisons in its trials87
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 |
|---|---|---|---|---|---|
| Dexpramipexole vs Placebo: deaths from any cause | 97 of 474 | 108 of 468 | 942 | NCT01281189 | Trial comparisons |
| Dexpramipexole vs Placebo: serious adverse events | 225 of 474 | 233 of 468 | 942 | NCT01281189 | Trial comparisons |
| Ceftriaxone vs Placebo: serious adverse events | 276 of 340 | 125 of 173 | 513 | NCT00349622 | Trial comparisons |
| Olesoxime vs Placebo Comparator: deaths from any cause | 79 of 259 | 80 of 253 | 512 | NCT00868166 | Trial comparisons |
| Olesoxime vs Placebo Comparator: serious adverse events | 68 of 259 | 65 of 253 | 512 | NCT00868166 | Trial comparisons |
| Levosimendan vs Placebo for Levosimendan: deaths from any cause | 27 of 326 | 17 of 166 | 492 | NCT03505021 | Trial comparisons |
| Levosimendan vs Placebo for Levosimendan: serious adverse events | 89 of 326 | 45 of 166 | 492 | NCT03505021 | Trial comparisons |
| Edaravone 105 mg (Once Daily) vs Edaravone 105mg (2 Weeks On/Off ): deaths from any cause | 9 of 192 | 16 of 192 | 384 | NCT04569084 | Trial comparisons |
| Edaravone 105 mg (Once Daily) vs Edaravone 105mg (2 Weeks On/Off ): serious adverse events | 52 of 192 | 55 of 191 | 383 | NCT04569084 | Trial comparisons |
| Glatiramer Acetate 40mg vs Placebo: deaths from any cause | 22 of 184 | 17 of 182 | 366 | NCT00326625 | Trial comparisons |
1 to 10 of 87
See all 87 comparisons in its trialsPapers from its trials139
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_0004976
GET https://conductscience.org/api/diseases/v1/MONDO_0004976/drugs?page=2