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granulomatous amebic encephalitis - Clinical Profile, Genes & Research | Kisho | Kisho

Rare Disease

granulomatous amebic encephalitis

MONDO:0000291

Also known as:granulomatous amoebic encephalitisAcanthamoeba encephalitisAcanthamoeba granulomatous encephalitis

Overview

Granulomatous amebic encephalitis is a life-threatening infection of the brain caused by the free-living amoebae Acanthamoeba spp., Balamuthia mandrillaris and Sappinia pedata. Acanthamoeba species, are commonly found in lakes, swimming pools, tap water, and heating and air conditioning units. The disease affects immunocompromised people and is very serious. Symptoms include mental status changes, loss of coordination, fever, muscular weakness or partial paralysis affecting one side of the body, double vision, sensitivity to light and other neurologic problems. The diagnosis is difficult and is often made at advanced stages. Tests useful in the diagnosis include brain scans, biopsies, or spinal taps and in disseminated disease, biopsy of the involved sites and testing by the laboratory experts. Early diagnosis is important for the prognosis. No single drug is effective; hence multiple antibiotics are needed for successful treatment. A combination of surgical and medical interventions involving multiple specialty experts is required to prevent death and morbidity in survivors.

TreatmentsNo approved treatments · 1 in pipeline

Diagnosis

Biomarker and diagnostic research for granulomatous amebic encephalitis 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 granulomatous amebic encephalitis. An additional 1 compound holds orphan drug designation.

Orphan Drug Designations

While no drugs are FDA-approved specifically for granulomatous amebic encephalitis, 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 granulomatous amebic encephalitis. 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 granulomatous amebic encephalitis.

81 publications have been identified in PubMed for granulomatous amebic encephalitis. Research spans Case Report / Case Series (31%), Gene Therapy / Novel Therapeutics (19%), and Basic Science / Preclinical (17%).

Research Type	Count	% of Total
Patient case studies	25	31%

Data assembled from 3 of 12 sources · Last updated Sep 18, 2026, 12:04 AM UTC

MONDOHPOClinicalTrials.govFDAPubMedHGNCClinVarClinGenGeneReviewsOrphanetOMIMRUSP

Medical References

External Resources

GARD

Genetic and Rare Diseases Info Center

GARD:12651

Questions for Your Care Team

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Kisho

granulomatous amebic encephalitis

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

Latest News & Developments

Granulomatous amoebic encephalitis: pathogenesis, diagnostic advances, therapeutic challenges, and emerging treatment strategies.

granulomatous amebic encephalitis

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

Latest News & Developments

Granulomatous amoebic encephalitis: pathogenesis, diagnostic advances, therapeutic challenges, and emerging treatment strategies.