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nondystrophic myotonia (NDM) - Clinical Profile, Genes & Research | Kisho | Kisho

Rare Disease

nondystrophic myotonia

MONDO:0054869

Also known as:NDM

Overview

TreatmentsNo approved treatments · 1 in pipeline

Diagnosis

Biomarker and diagnostic research for nondystrophic myotonia 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.

No approved treatments are currently available for nondystrophic myotonia. An additional 1 compound holds orphan drug designation.

Orphan Drug Designations

While no drugs are FDA-approved specifically for nondystrophic myotonia, some of the following designated compounds may be used off-label in clinical practice. Treatment decisions should be made in consultation with a specialist familiar with this condition.

The following drugs have received orphan drug designation from the FDA for nondystrophic myotonia. Orphan designation reflects regulatory interest and does not indicate approval for treatment.

Brand Name	Generic Name	Sponsor

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.

No clinical trials have been registered for nondystrophic myotonia.

12 publications have been identified in PubMed for nondystrophic myotonia. Research spans Clinical Trial Publication (25%), Review / Meta-Analysis (17%), and Case Report / Case Series (17%).

Research Type	Count	% of Total
Clinical study results	3	25%

Data assembled from 3 of 12 sources · Last updated Sep 20, 2026, 4:02 PM UTC

MONDOHPOClinicalTrials.govFDAPubMedHGNCClinVarClinGenGeneReviewsOrphanetOMIMRUSP

Medical References

External Resources

GARD

Genetic and Rare Diseases Info Center

GARD:9852

Questions for Your Care Team

→What is the current development status of mexiletine, and when might it become available?
→Are there specialists or centers of excellence for this condition in my area?
→Is there a patient registry I should consider joining?

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Kisho

nondystrophic myotonia

Overview

Diagnosis

Treatment

Orphan Drug Designations

Research Landscape

Medical References

External Resources

GARD

Questions for Your Care Team

Questions for Your Care Team

Clinical Trials

Recent Publications

nondystrophic myotonia

Overview

Diagnosis

Treatment

Orphan Drug Designations

Research Landscape

Medical References

External Resources

GARD

Related Diseases

Questions for Your Care Team

Questions for Your Care Team

Clinical Trials

Recent Publications