Neurofaciodigitorenal syndrome

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ORPHA:2673OMIM:256690Q87.8
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8Treatment centers

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Overview

Neurofaciodigitorenal syndrome (also sometimes called NFDR syndrome) is a very rare condition that affects multiple parts of the body at the same time, including the brain and nervous system, the face, the fingers and toes, and the kidneys. Because it touches so many different body systems, it is considered a 'multi-system' syndrome. The exact number of people affected worldwide is not well known, as only a very small number of cases have ever been reported in medical literature. People with this syndrome may have distinctive facial features that are noticeable from birth or early infancy, along with differences in the shape or number of fingers and toes. Kidney problems can range from mild to more serious and may need ongoing monitoring. Neurological features, such as intellectual disability or developmental delays, are also commonly seen and can affect learning, communication, and daily functioning. There is currently no cure for neurofaciodigitorenal syndrome. Treatment focuses on managing each symptom as it appears, which may involve a team of specialists including neurologists, kidney doctors, and developmental pediatricians. Early intervention programs and therapies such as speech, occupational, and physical therapy can make a meaningful difference in quality of life.

Also known as:

Key symptoms:

Intellectual disability or learning difficultiesDelayed development in talking and walkingDistinctive facial features such as a broad or flat nose, widely spaced eyes, or low-set earsAbnormal fingers or toes (extra digits, fused digits, or unusual shape)Kidney abnormalities or reduced kidney functionShort stature or slow growthHearing lossSeizuresFeeding difficulties in infancyMuscle weakness or low muscle tone

Clinical phenotype terms (30)— hover any for plain English
Abnormal antitragus morphologyHP:0009896Abnormal tragus morphologyHP:0009912Hypoplasia of the premaxillaHP:0010650Abnormal oral mucosa morphologyHP:0011830Abnormality of the philtrumHP:0000288
Inheritance

Autosomal recessive

Passed on when both parents carry the same gene change; often skips generations

Age of Onset

Neonatal

Begins at or shortly after birth (first 4 weeks)

Orphanet ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Neurofaciodigitorenal syndrome.

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No actively recruiting trials found for Neurofaciodigitorenal syndrome at this time.

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

Search ClinicalTrials.gov ↗Join the Neurofaciodigitorenal syndrome community →

No specialists are currently listed for Neurofaciodigitorenal 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 Neurofaciodigitorenal syndrome.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Neurofaciodigitorenal syndrome

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Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

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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.What genetic tests should we do to confirm the diagnosis and understand the cause?,How often should my child's kidney function be checked, and what signs of worsening should I watch for at home?,What therapies (speech, occupational, physical) should we start right away, and how do we access them?,Are there any clinical trials or research studies we could participate in?,What is the likely progression of this condition, and what should we plan for as my child gets older?,Are other family members at risk, and should they be tested?,What support services or patient organizations exist that could help our family?

Common questions about Neurofaciodigitorenal syndrome

What is Neurofaciodigitorenal syndrome?

Neurofaciodigitorenal syndrome (also sometimes called NFDR syndrome) is a very rare condition that affects multiple parts of the body at the same time, including the brain and nervous system, the face, the fingers and toes, and the kidneys. Because it touches so many different body systems, it is considered a 'multi-system' syndrome. The exact number of people affected worldwide is not well known, as only a very small number of cases have ever been reported in medical literature. People with this syndrome may have distinctive facial features that are noticeable from birth or early infancy, al

How is Neurofaciodigitorenal syndrome inherited?

Neurofaciodigitorenal syndrome follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Neurofaciodigitorenal syndrome typically begin?

Typical onset of Neurofaciodigitorenal syndrome is neonatal. Age of onset can vary across affected individuals.