Patent ductus arteriosus-bicuspid aortic valve-hand anomalies syndrome

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ORPHA:228190OMIM:604381Q87.2
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1FDA treatments8Treatment centers

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Overview

Patent ductus arteriosus-bicuspid aortic valve-hand anomalies syndrome is an extremely rare genetic condition that affects the heart and hands. The name describes its three main features. A patent ductus arteriosus (PDA) is a heart defect present at birth where a blood vessel called the ductus arteriosus, which normally closes shortly after birth, stays open. This can cause abnormal blood flow between the two major arteries leaving the heart. A bicuspid aortic valve means the aortic valve has two flaps instead of the normal three, which can affect how well the valve opens and closes. Hand anomalies in this syndrome can include differences in the shape, size, or structure of the fingers or hands. Because this syndrome involves both heart defects and limb differences, it is classified as a multiple congenital anomaly syndrome. The condition is typically identified at birth or in early infancy when heart murmurs or hand differences are noticed. Treatment focuses on managing the heart defects, which may require medication or surgery to close the PDA and monitor or repair the bicuspid aortic valve. Hand anomalies may also require surgical correction or occupational therapy depending on their severity. Long-term follow-up with a cardiologist is important to watch for complications such as valve narrowing or leaking over time.

Also known as:

Key symptoms:

Heart murmur detected at birthPatent ductus arteriosus (a blood vessel in the heart that stays open after birth)Bicuspid aortic valve (the aortic valve has two flaps instead of three)Abnormalities of the fingers or handsShort fingers or toesFifth finger curving inward (clinodactyly)Breathing difficulties in infancyPoor feeding in newbornsFatigue or tiring easilyDelayed growth in some cases

Clinical phenotype terms (8)— hover any for plain English
Pseudocoarctation of the aortaHP:0005295Abnormal hand morphologyHP:0005922Short 5th metacarpalHP:0010047Short digitHP:0011927
Inheritance

Autosomal dominant

Passed on from just one parent; each child has about a 50% chance of inheriting it

Age of Onset

Neonatal

Begins at or shortly after birth (first 4 weeks)

Orphanet ↗OMIM ↗NORD ↗

FDA & Trial Timeline

1 event
Oct 2016

VERMOX 500 mg chewable tablets: FDA approved

treatment of patients one year of age and older withgastrointestinal infections caused by Ascaris lumbricoides (roundworm) and Trichuris trichiura (whipworm)

FDAcompleted

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

1 available

VERMOX 500 mg chewable tablets

mebendazole· Janssen Pharmaceutical Research & Development, LLCOrphan Drug

treatment of patients one year of age and older withgastrointestinal infections caused by Ascaris lumbricoides (roundworm) and Trichuris trichiura (whipworm)

No actively recruiting trials found for Patent ductus arteriosus-bicuspid aortic valve-hand anomalies syndrome at this time.

New trials open frequently. Follow this disease to get notified.

Search ClinicalTrials.gov ↗Join the Patent ductus arteriosus-bicuspid aortic valve-hand anomalies syndrome community →

No specialists are currently listed for Patent ductus arteriosus-bicuspid aortic valve-hand anomalies syndrome.

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 Patent ductus arteriosus-bicuspid aortic valve-hand anomalies syndrome.

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Community

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Latest news about Patent ductus arteriosus-bicuspid aortic valve-hand anomalies syndrome

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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.

Questions for your doctor

Bring these to your next appointment

  • Q1.How severe is my child's patent ductus arteriosus, and will it need surgery or can it be treated with medication?,How often should the bicuspid aortic valve be monitored, and what signs of worsening should I watch for?,Are there any activity or exercise restrictions my child should follow?,Would genetic testing help us understand the cause and the chance of this happening in future pregnancies?,Does my child need antibiotics before dental procedures to prevent heart infections?,What therapies are available to help with hand function?,Should other family members be screened for heart defects?

Common questions about Patent ductus arteriosus-bicuspid aortic valve-hand anomalies syndrome

What is Patent ductus arteriosus-bicuspid aortic valve-hand anomalies syndrome?

Patent ductus arteriosus-bicuspid aortic valve-hand anomalies syndrome is an extremely rare genetic condition that affects the heart and hands. The name describes its three main features. A patent ductus arteriosus (PDA) is a heart defect present at birth where a blood vessel called the ductus arteriosus, which normally closes shortly after birth, stays open. This can cause abnormal blood flow between the two major arteries leaving the heart. A bicuspid aortic valve means the aortic valve has two flaps instead of the normal three, which can affect how well the valve opens and closes. Hand anom

How is Patent ductus arteriosus-bicuspid aortic valve-hand anomalies syndrome inherited?

Patent ductus arteriosus-bicuspid aortic valve-hand anomalies syndrome follows a autosomal dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Patent ductus arteriosus-bicuspid aortic valve-hand anomalies syndrome typically begin?

Typical onset of Patent ductus arteriosus-bicuspid aortic valve-hand anomalies syndrome is neonatal. Age of onset can vary across affected individuals.

What treatment and support options exist for Patent ductus arteriosus-bicuspid aortic valve-hand anomalies syndrome?

1 patient support program are currently tracked on UniteRare for Patent ductus arteriosus-bicuspid aortic valve-hand anomalies syndrome. See the treatments and support programs sections for copay assistance, eligibility, and contact details.