Disease · EFO_0020094

Lambert-Eaton myasthenic syndrome

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. Lambert-Eaton myasthenic syndrome. Diseases, record EFO_0020094. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/EFO_0020094

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: Lambert-Eaton myasthenic syndrome; Open Targets
Open Targets ID: EFO_0020094; Open Targets
Other names: LEMS; Open Targets
MeSH: D015624; MeSH
Orphanet: ORPHA:43393; Orphanet
Highest stage of its drugs: Approval, for 2 of its 2 drugs, from ClinicalTrials.gov, EMA Human Drugs and PMDA; Open Targets

Drugs2

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
AMIFAMPRIDINE	CHEMBL354077	Small molecule	Approval	ClinicalTrials.gov and EMA Human Drugs	12	10	1	Drugs and targets
AMIFAMPRIDINE PHOSPHATE	CHEMBL3301611	Small molecule	Approval	ClinicalTrials.gov and PMDA	4	3	3	Drugs and targets

Targets40

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
KCNA1	Voltage-gated potassium channel	2	Voltage-gated potassium channel blocker	Approval	https://conductscience.org/library/genes/3736	Drugs and targets
KCNA10	Voltage-gated potassium channel	2	Voltage-gated potassium channel blocker	Approval	https://conductscience.org/library/genes/3744	Drugs and targets
KCNA2	Voltage-gated potassium channel	2	Voltage-gated potassium channel blocker	Approval	https://conductscience.org/library/genes/3737	Drugs and targets
KCNA3	Voltage-gated potassium channel	2	Voltage-gated potassium channel blocker	Approval	https://conductscience.org/library/genes/3738	Drugs and targets
KCNA4	Voltage-gated potassium channel	2	Voltage-gated potassium channel blocker	Approval	https://conductscience.org/library/genes/3739	Drugs and targets
KCNA5	Voltage-gated potassium channel	2	Voltage-gated potassium channel blocker	Approval	https://conductscience.org/library/genes/3741	Drugs and targets
KCNA6	Voltage-gated potassium channel	2	Voltage-gated potassium channel blocker	Approval	https://conductscience.org/library/genes/3742	Drugs and targets
KCNA7	Voltage-gated potassium channel	2	Voltage-gated potassium channel blocker	Approval	https://conductscience.org/library/genes/3743	Drugs and targets
KCNB1	Voltage-gated potassium channel	2	Voltage-gated potassium channel blocker	Approval	https://conductscience.org/library/genes/3745	Drugs and targets
KCNB2	Voltage-gated potassium channel	2	Voltage-gated potassium channel blocker	Approval	https://conductscience.org/library/genes/9312	Drugs and targets

1 to 10 of 40

See all 40 targets

Trials4

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. 9 more are not shown, since the checks do not confirm them.

Trial	Title	Phase	Participants	Results first posted	Conditions	Drugs	Linked by	Source
NCT01377922	A Phase 3 Study of Amifampridine Phosphate in Patients With Lambert Eaton Myasthenic Syndrome (LEMS)	Phase 3	38	2018-01-04	Lambert Eaton Myasthenic Syndrome	AMIFAMPRIDINE PHOSPHATE	Its record names the disease	ClinicalTrials.gov
NCT01511978	Effectiveness of 3,4-Diaminopyridine in Lambert-Eaton Myasthenic Syndrome	Phase 2	32	2017-07-11	Lambert-Eaton Myasthenic Syndrome, Eaton-Lambert Myasthenic Syndrome	AMIFAMPRIDINE	Its record names the disease	ClinicalTrials.gov
NCT02970162	Phase 3 Study to Evaluate Efficacy of Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS)	Phase 3	26	2018-12-24	Lambert-Eaton Myasthenic Syndrome	AMIFAMPRIDINE PHOSPHATE	Its record names the disease	ClinicalTrials.gov
NCT02189720	Expanded Access Study Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS),Congenital Myasthenic Syndrome				Congenital Myasthenic Syndrome, Lambert-Eaton Myasthenic Syndrome, Nystagmus, Acquired	AMIFAMPRIDINE PHOSPHATE	Its record names the disease	ClinicalTrials.gov

Comparisons in its trials8

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 2 and Part 3 Placebo vs Part 2 and Part 3 Amifampridine Phosphate: deaths from any cause	0 of 22	0 of 16	38	NCT01377922	Trial comparisons
Part 2 and Part 3 Placebo vs Part 2 and Part 3 Amifampridine Phosphate: serious adverse events	0 of 22	0 of 16	38	NCT01377922	Trial comparisons
Continuous 3,4-Diaminopyridine (3,4-DAP) vs 3,4-DAP Taper to Placebo: Number of Participants With 30% or More Deterioration in Triple Timed Up & Go (3TUG) Test, Compared to Time-matched Baseline	0 of 14	13 of 18	32	NCT01511978	Trial comparisons
Continuous 3,4-Diaminopyridine (3,4-DAP) vs 3,4-DAP Taper to Placebo: deaths from any cause	0 of 14	0 of 18	32	NCT01511978	Trial comparisons
Continuous 3,4-Diaminopyridine (3,4-DAP) vs 3,4-DAP Taper to Placebo: serious adverse events	0 of 14	1 of 18	32	NCT01511978	Trial comparisons
Amifampridine Phosphate vs Placebo (for Amifampridine Phosphate): Triple Timed Up and Go Walk Test (3TUG)	1 of 13	8 of 13	26	NCT02970162	Trial comparisons
Amifampridine Phosphate vs Placebo (for Amifampridine Phosphate): deaths from any cause	0 of 13	0 of 13	26	NCT02970162	Trial comparisons
Amifampridine Phosphate vs Placebo (for Amifampridine Phosphate): serious adverse events	0 of 13	0 of 13	26	NCT02970162	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/EFO_0020094

GET https://conductscience.org/api/diseases/v1/EFO_0020094/targets?page=2

API reference For AI assistants