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pediatric multiple sclerosis - Clinical Profile, Genes & Research | Kisho | Kisho

Rare Disease

pediatric multiple sclerosis

MONDO:0018784

Also known as:MS paediatricMS pediatricmultiple sclerosis, paediatric

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Overview

Pediatric multiple sclerosis (MS) is a rare multiple sclerosis variant characterized by the onset of multiple sclerosis (i.e. one or multiple episodes of clinical CNS symptoms consistent with acquired CNS demyelination, with radiologically proven dissemination of inflammatory lesions in space and time, following exclusion of other disorders) before the age of 18 years old. Pediatric MS patients present a predominantly relapsing-remitting course with first attack usually consisting of optic neuritis, transverse myelitis, acute disseminated encephalomyelitis and monofocal or polyfocal neurological deficits. A high burden of T2-hyperintense lesions on initial MRI, primarily of the supratentorial region and/or of the cervical spinal cord, has been reported.

TreatmentsNo approved treatments · 4 in pipeline

Clinical Trials2 registered

Patient Orgs1 organizations · Tanner Foundation for Multiple Sclerosis

Diagnosis

Biomarker and diagnostic research for pediatric multiple sclerosis 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 pediatric multiple sclerosis. An additional 4 compounds hold orphan drug designation.

Orphan Drug Designations

While no drugs are FDA-approved specifically for pediatric multiple sclerosis, 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 pediatric multiple sclerosis. 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.

2 clinical trials registered. Interventions under study include other interventions. Pipeline includes 1 NA. Research is primarily sponsored by academic and government institutions.

175 publications have been identified in PubMed for pediatric multiple sclerosis. Research spans Epidemiology / Natural History (26%), Review / Meta-Analysis (21%), and Basic Science / Preclinical (14%).

Research Type	Count	% of Total
Disease patterns and progression	37

Data assembled from 5 of 12 sources · Last updated Sep 19, 2026, 12:48 AM UTC

MONDOHPOClinicalTrials.govFDAPubMedHGNCClinVarClinGenGeneReviewsOrphanetOMIMRUSP

Patient Support

Patient Advocacy Groups

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Tanner Foundation for Multiple SclerosisPrimary Focus

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:477738

GARD

Genetic and Rare Diseases Info Center

GARD:10443

Questions for Your Care Team

→Are any of the 2 active clinical trials a good fit for my situation?
→With 4 compounds in the orphan drug pipeline, which are furthest along in development and which clinical trials should I be aware of?
→Are there specialists or centers of excellence for this condition in my area?
→How can I connect with Tanner Foundation for Multiple Sclerosis and other patient organizations for support?

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pediatric multiple sclerosis

Overview

Diagnosis

Treatment

Orphan Drug Designations

Research Landscape

Patient Support

Patient Advocacy Groups

Medical References

External Resources

Orphanet

GARD

Related Diseases

Questions for Your Care Team

Questions for Your Care Team

Clinical Trials

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