Overview
Also known as:
Clinical phenotype terms— hover any for plain English:
FDA & Trial Timeline
10 eventsMayo Clinic — EARLY_PHASE1
National Institute of Neurological Disorders and Stroke (NINDS) — NA
University Hospital, Basel, Switzerland — NA
University of California, Los Angeles — PHASE2
Mayo Clinic
Hospices Civils de Lyon
Huashan Hospital — NA
Isturisa: FDA approved
ISTURISA is a cortisol synthesis inhibitor indicated for the treatment of adult patients with Cushing®s disease for whom pituitary surgery is not an option or has not been curative.
Centre Hospitalier Universitaire de Nīmes
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
2 availableIsturisa
ISTURISA is a cortisol synthesis inhibitor indicated for the treatment of adult patients with Cushing�s disease for whom pituitary surgery is not an option or has not been curative.
Signifor
SIGNIFOR� LAR is indicated for the treatment of patients with Cushing�s disease for whom pituitary surgery is not an option or has not been curative.
Rare Disease Specialist
Treatment Centers
8 centersBaylor College of Medicine Rare Disease Center ↗
Baylor College of Medicine
📍 Houston, TX
🏥 NORDStanford Medicine Rare Disease Center ↗
Stanford Medicine
📍 Stanford, CA
🔬 UDNNIH Clinical Center Undiagnosed Diseases Program ↗
National Institutes of Health
📍 Bethesda, MD
🔬 UDNUCLA UDN Clinical Site ↗
UCLA Health
📍 Los Angeles, CA
🔬 UDNBaylor College of Medicine UDN Clinical Site ↗
Baylor College of Medicine
📍 Houston, TX
🔬 UDNHarvard/MGH UDN Clinical Site ↗
Massachusetts General Hospital
📍 Boston, MA
🏥 NORDMayo Clinic Center for Individualized Medicine ↗
Mayo Clinic
📍 Rochester, MN
👤 Mayo Clinic Center for Individualized Medicine
🏥 NORDUCLA Rare Disease Day Program ↗
UCLA Health
📍 Los Angeles, CA
Financial Resources
2 resourcesTravel Grants
No travel grants are currently matched to Cushing disease.
Community
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Start the conversation →Latest news about Cushing disease
Disease timeline:
New recruiting trial: A Trial of Lu AG13909 in Adult Participants With Cushing's Disease
A new clinical trial is recruiting patients for Cushing disease
New recruiting trial: Long-Term Follow-Up of Survivors of Pediatric Cushing Disease
A new clinical trial is recruiting patients for Cushing disease
New recruiting trial: DDAVP for Pituitary Adenoma
A new clinical trial is recruiting patients for Cushing disease
New recruiting trial: Evaluation of Intraoperative Contrast Enhanced Ultrasound for the Identification of Pituitary Adenoma in Cushing's Disease Compared to Other Pituitary Tumors
A new clinical trial is recruiting patients for Cushing disease
New recruiting trial: Evaluation of Positron Emission Tomography (PET) With [18F]FET for the Detection of ACTH-Secreting Corticotroph Pituitary Neuroendocrine Tumors.
A new clinical trial is recruiting patients for Cushing disease
New recruiting trial: Multicenter Study of Seliciclib (R-roscovitine) for Cushing Disease
A new clinical trial is recruiting patients for Cushing disease
New recruiting trial: Fimepinostat, Combination HDAC and Pi3-kinase Inhibitor Tumor-Directed Therapy for Cushing Disease
A new clinical trial is recruiting patients for Cushing disease
Caregiver Resources
NORD Caregiver Resources
Support, advocacy, and financial assistance for caregivers of rare disease patients.
Mental Health Support
Rare disease caregiving can be isolating. Connect with counseling and peer support.
Family & Caregiver Grants
Financial assistance programs specifically for caregivers of rare disease patients.
Social Security Disability
Learn how rare disease patients may qualify for SSDI/SSI benefits.
Common questions about Cushing disease
Are there clinical trials for Cushing disease?
Yes — 9 recruiting clinical trials are currently listed for Cushing disease on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.
Which specialists treat Cushing disease?
25 specialists and care centers treating Cushing disease are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.
What treatment and support options exist for Cushing disease?
3 patient support programs are currently tracked on UniteRare for Cushing disease. See the treatments and support programs sections for copay assistance, eligibility, and contact details.