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Lambert-Eaton myasthenic syndrome - Clinical Profile, Treatments & Trials | Kisho | Kisho

Rare Disease

Lambert-Eaton myasthenic syndrome

MONDO:0018556

Also known as:Eaton Lambert syndromeLEMSLambert Eaton syndrome

Find Clinical TrialsTrials

Overview

Lambert-Eaton myasthenic syndrome (LEMS) is an autoimmune, presynaptic disorder of neuromuscular transmission characterized by fluctuating muscle weakness and autonomic dysfunction frequently associated with small-cell lung cancer (SCLC).

Treatments1 FDA approved

Clinical Trials5 registered · 2 recruiting

Patient Orgs1 organizations · The Lambert-Eaton LEMS Family Association

Diagnosis

Biomarker and diagnostic research for Lambert-Eaton myasthenic syndrome has been reported in the published literature.

Treatment

Compiled from FDA-approved drug records, orphan drug designations, and FDA Adverse Event Reporting System (FAERS) safety data. GeneReviews management guidance is included where available.

1 FDA-approved treatment is available for Lambert-Eaton myasthenic syndrome, including AMIFAMPRIDINE PHOSPHATE (FIRDAPSE, approved 2018). An additional 2 compounds hold orphan drug designation.

Approved Therapies

Brand Name	Generic Name	Mechanism	Approved	Market Status

Prognosis

Estimated prevalence: 1-9 in 1,000,000 (Rare).

Research Landscape

Sourced from ClinicalTrials.gov and PubMed. Publication types are classified using NLM-curated article metadata; research type breakdowns reflect the nature of published work for this disease.

5 clinical trials registered, 2 recruiting. Interventions under study include other interventions, medical devices, drug therapy, and procedural interventions. Pipeline includes 1 PHASE2, 2 NA. Research is primarily sponsored by academic and government institutions.

NCT ID	Title	Phase	Sponsor	Status
[NCT07075627](https://clinicaltrials.gov/study/NCT07075627)

Data assembled from 5 of 12 sources · Last updated Sep 19, 2026, 9:41 PM UTC

MONDOHPOClinicalTrials.govFDAPubMedHGNCClinVarClinGenGeneReviewsOrphanetOMIMRUSP

Patient Support

Financial & Access Support (External Programs)

View all programs →

Lambert-Eaton Myasthenic Syndrome Fund

Premium/CopayClosedOperated by HealthWell Foundation

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LEMS Medical Assistance

Medical AssistanceCurrently AcceptingOperated by National Organization for Rare Disorders

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Lambert-Eaton Myasthenic Syndrome (LEMS) Premium Copay Assistance

Premium/CopayCurrently AcceptingOperated by National Organization for Rare Disorders

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Program availability and eligibility requirements are set by each foundation. Contact them directly to learn more about your options.

Patient Advocacy Groups

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The Lambert-Eaton LEMS Family AssociationPrimary Focus

USWebsite ↗

Patient Advocacy Groups (PAGs) provide support, resources, and community for patients and caregivers.

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Medical References

External Resources

Orphanet

European rare disease database

Orphanet:43393

GARD

Genetic and Rare Diseases Info Center

GARD:6851

Questions for Your Care Team

→What are the benefits and potential side effects of FIRDAPSE for managing Lambert-Eaton myasthenic syndrome?
→Are any of the 5 active clinical trials a good fit for my situation?
→Are there specialists or centers of excellence for this condition in my area?
→How can I connect with The Lambert-Eaton LEMS Family Association and other patient organizations for support?

← Back to All Diseases

Brand Name	Generic Name	Sponsor	Designated	Exclusivity End	Designation Status
3,4 diaminopyridine and choline bitartrate	3,4 diaminopyridine and choline bitartrate	MS Therapeutics Limited	2010	—	Designated
Dynamine	Dynamine	Mayo Foundation	1990	—	Designated

Research Type	Count	% of Total
Patient case studies	36	44%
Research summaries	27	33%
Disease patterns and progression	7	9%
Testing and diagnosis research	5	6%
Other research	3	4%
Clinical study results	2	2%
Laboratory research	2	2%

Kisho

Lambert-Eaton myasthenic syndrome

Overview

Diagnosis

Treatment

Approved Therapies

Prognosis

Research Landscape

Patient Support

Financial & Access Support (External Programs)

Patient Advocacy Groups

Medical References

External Resources

Orphanet

GARD

Questions for Your Care Team

Questions for Your Care Team

Orphan Drug Designations

Clinical Trials

Recent Publications

Latest News & Developments

Multidisciplinary Management of Lymphangioleiomyomatosis Presenting With Severe Bilateral Pneumothorax During Pregnancy: A Case Report.

Longitudinal overview of symptomatic and immunosuppressive drugs in LEMS.

Lambert-Eaton myasthenic syndrome

Overview

Diagnosis

Treatment

Approved Therapies

Prognosis

Research Landscape

Patient Support

Financial & Access Support (External Programs)

Patient Advocacy Groups

Medical References

External Resources

Orphanet

GARD

Related Diseases

Questions for Your Care Team

Questions for Your Care Team

Orphan Drug Designations

Clinical Trials

Recent Publications

Latest News & Developments

Multidisciplinary Management of Lymphangioleiomyomatosis Presenting With Severe Bilateral Pneumothorax During Pregnancy: A Case Report.

Longitudinal overview of symptomatic and immunosuppressive drugs in LEMS.