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superficial siderosis - Clinical Profile, Genes & Research | Kisho | Kisho

Rare Disease

superficial siderosis

MONDO:0016594

Also known as:hemosiderosis of the central nervous systemsuperficial hemosiderosis of the CNSsuperficial hemosiderosis of the central nervous system

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Overview

Superficial siderosis is a rare neurologic disease characterized by progressive sensorineural hearing loss, cerebellar ataxia, pyramidal signs, and neuroimaging findings revealing hemosiderin deposits in the spinal and cranial leptomeninges and subpial layer. The disease progresses slowly and patients may present with mild cognitive impairment, nystagmus, dysmetria, spasticity, dysdiadochokinesia, dysarthria, hyperreflexia, and Babinski signs. Additional features reported include dementia, urinary incontinence, anosmia, ageusia, and anisocoria.

TreatmentsNo approved treatments · 1 in pipeline

Clinical Trials2 registered · 1 recruiting

Patient Orgs1 organizations · Superficial Siderosis Research Alliance, Inc.

Diagnosis

Biomarker and diagnostic research for superficial siderosis 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 superficial siderosis. An additional 1 compound holds orphan drug designation.

Orphan Drug Designations

While no drugs are FDA-approved specifically for superficial siderosis, 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 superficial siderosis. Orphan designation reflects regulatory interest and does not indicate approval for treatment.

Brand Name	Generic Name	Sponsor

Prognosis

Estimated prevalence: Unknown (Unknown prevalence).

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, 1 recruiting. Interventions under study include other interventions. Pipeline includes 1 NA. Research is sponsored by a mix of industry and academic institutions.

165 publications have been identified in PubMed for superficial siderosis. Research spans Case Report / Case Series (31%), Review / Meta-Analysis (22%), and Epidemiology / Natural History (22%).

Research Type	Count	% of Total
Patient case studies	51

Data assembled from 5 of 12 sources · Last updated Sep 19, 2026, 7:49 AM UTC

MONDOHPOClinicalTrials.govFDAPubMedHGNCClinVarClinGenGeneReviewsOrphanetOMIMRUSP

Patient Support

Patient Advocacy Groups

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Superficial Siderosis Research Alliance, Inc.Primary 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:247245

GARD

Genetic and Rare Diseases Info Center

GARD:9484

Questions for Your Care Team

→Are any of the 2 active clinical trials a good fit for my situation?
→What is the current development status of deferiprone, and when might it become available?
→Are there specialists or centers of excellence for this condition in my area?
→How can I connect with Superficial Siderosis Research Alliance, Inc. and other patient organizations for support?

← Back to All Diseases

Kisho

superficial siderosis

Overview

Diagnosis

Treatment

Orphan Drug Designations

Prognosis

Research Landscape

Patient Support

Patient Advocacy Groups

Medical References

External Resources

Orphanet

GARD

Questions for Your Care Team

Questions for Your Care Team

Clinical Trials

Recent Publications

Latest News & Developments

Superficial siderosis caused by a tiny dural defect localized by an isodense crossing point on CT myelography: illustrative case.

Giant cauda equina neuroendocrine tumor with superficial siderosis: illustrative case.

superficial siderosis

Overview

Diagnosis

Treatment

Orphan Drug Designations

Prognosis

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

Recent Publications

Latest News & Developments

Superficial siderosis caused by a tiny dural defect localized by an isodense crossing point on CT myelography: illustrative case.

Giant cauda equina neuroendocrine tumor with superficial siderosis: illustrative case.