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endogenous Cushing syndrome - Clinical Profile, Genes & Research | Kisho | Kisho

Rare Disease

endogenous Cushing syndrome

MONDO:0957431

Also known as:endogenous CS

Find Clinical TrialsTrials

Overview

Any Cushing syndrome caused by body's prolonged overproduction of cortisol that can be dependent on or independent of adrenocorticotropic hormone (ACTH).

TreatmentsNo approved treatments · 3 in pipeline

Clinical Trials2 registered · 1 recruiting

Diagnosis

Biomarker and diagnostic research for endogenous Cushing 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.

No approved treatments are currently available for endogenous Cushing syndrome. An additional 3 compounds hold orphan drug designation.

Orphan Drug Designations

While no drugs are FDA-approved specifically for endogenous Cushing syndrome, 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 endogenous Cushing syndrome. 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, 1 recruiting. Interventions under study include drug therapy. Pipeline includes 1 PHASE2. Research is sponsored by a mix of industry and academic institutions.

54 publications have been identified in PubMed for endogenous Cushing syndrome. Research spans Epidemiology / Natural History (30%), Case Report / Case Series (19%), and Diagnostic / Biomarker (15%).

Research Type	Count	% of Total
Disease patterns and progression	16

Data assembled from 5 of 12 sources · Last updated Sep 20, 2026, 5:49 AM UTC

MONDOHPOClinicalTrials.govFDAPubMedHGNCClinVarClinGenGeneReviewsOrphanetOMIMRUSP

Medical References

External Resources

Orphanet

European rare disease database

Orphanet:641613

Questions for Your Care Team

→Are any of the 2 active clinical trials a good fit for my situation?
→With 3 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?
→Is there a patient registry I should consider joining?

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Kisho

endogenous Cushing syndrome

Overview

Diagnosis

Treatment

Orphan Drug Designations

Research Landscape

Medical References

External Resources

Orphanet

Questions for Your Care Team

Questions for Your Care Team

Clinical Trials

Recent Publications

Latest News & Developments

[Need for Improvement in Healthcare Provision for Rare Endocrinological Diseases in Germany Using the Example Endogenous Cushing's Syndrome].

endogenous Cushing syndrome

Overview

Diagnosis

Treatment

Orphan Drug Designations

Research Landscape

Medical References

External Resources

Orphanet

Related Diseases

Questions for Your Care Team

Questions for Your Care Team

Clinical Trials

Recent Publications

Latest News & Developments

[Need for Improvement in Healthcare Provision for Rare Endocrinological Diseases in Germany Using the Example Endogenous Cushing's Syndrome].