Overview

Also known as:

Rare familial disorder with hypertrophic obstructive cardiomyopathy Rare familial disorder with hypertrophic subaortic stenosis

Orphanet ↗NORD ↗

Treatments

1 available

Sodium Phenylbutyrate

SODIUM PHENYLBUTYRATE TABLETS, 500 MG· ENDO USA, Inc.

indicated in patients with late-onset disease (partial enzymatic deficiency, presenting after the first month of life) who have a history of hyperammonemic encephalopathy

View Details →FDA Label ↗

Clinical Trials

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No actively recruiting trials found for Rare familial disorder with hypertrophic cardiomyopathy at this time.

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Specialists

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No specialists are currently listed for Rare familial disorder with hypertrophic cardiomyopathy.

View NORD Rare Disease Centers ↗Undiagnosed Disease Network ↗

Treatment Centers

8 centers

🏥 NORD

Baylor College of Medicine Rare Disease Center ↗

Baylor College of Medicine

📍 Houston, TX

🏥 NORD

Stanford Medicine Rare Disease Center ↗

Stanford Medicine

📍 Stanford, CA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program ↗

National Institutes of Health

📍 Bethesda, MD

🔬 UDN

UCLA UDN Clinical Site ↗

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site ↗

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site ↗

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine ↗

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🏥 NORD

UCLA Rare Disease Day Program ↗

UCLA Health

📍 Los Angeles, CA

Travel Grants

No travel grants are currently matched to Rare familial disorder with hypertrophic cardiomyopathy.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Rare familial disorder with hypertrophic cardiomyopathy

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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.