Genetic non-syndromic renal or urinary tract malformation

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Overview

Genetic non-syndromic renal or urinary tract malformation refers to a group of birth defects affecting the kidneys, ureters, bladder, or urethra that are caused by changes in a person's genes. The term 'non-syndromic' means these malformations occur on their own, without being part of a larger syndrome that affects other body systems. These malformations are also known as congenital anomalies of the kidney and urinary tract (CAKUT) when they have a genetic basis and appear in isolation. The types of malformations can vary widely. They may include kidneys that did not form properly (renal agenesis or dysplasia), kidneys that are in the wrong position (ectopic kidney), blockages in the urinary tract (obstruction), backward flow of urine from the bladder to the kidneys (vesicoureteral reflux), or duplicated collecting systems. Some people have mild forms that cause few or no symptoms, while others may develop serious kidney problems including chronic kidney disease or kidney failure. Many of these malformations are detected before birth on prenatal ultrasound or during childhood when a child develops urinary tract infections, difficulty urinating, or abnormal kidney function tests. Treatment depends on the specific type and severity of the malformation. Options range from careful monitoring and medications to surgical correction. Early detection and management are important to preserve kidney function and prevent complications like high blood pressure and kidney failure. Genetic counseling is recommended for affected families to understand the chance of the condition occurring in future children.

Key symptoms:

Kidney that did not develop properly or is missingSwelling of the kidney due to urine backup (hydronephrosis)Repeated urinary tract infectionsBackward flow of urine from bladder to kidneys (vesicoureteral reflux)Difficulty urinating or weak urine streamHigh blood pressureAbnormal kidney function on blood testsProtein or blood in the urineSlow growth or failure to thrive in childrenKidney that is in an unusual positionDuplicated kidney or collecting systemChronic kidney disease or kidney failureBedwetting or urinary incontinenceAbdominal or flank pain

Inheritance

Variable

Can be inherited in different ways depending on the underlying gene

Age of Onset

Neonatal

Begins at or shortly after birth (first 4 weeks)

Orphanet ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Genetic non-syndromic renal or urinary tract malformation.

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No actively recruiting trials found for Genetic non-syndromic renal or urinary tract malformation at this time.

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No specialists are currently listed for Genetic non-syndromic renal or urinary tract malformation.

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 Genetic non-syndromic renal or urinary tract malformation.

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Community

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Caregiver Resources

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Social Security Disability

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Questions for your doctor

Bring these to your next appointment

  • Q1.What specific type of kidney or urinary tract malformation does my child have, and how severe is it?,Should we pursue genetic testing, and if so, what genes will be tested?,What is the long-term outlook for kidney function, and what are the chances of needing dialysis or a transplant?,How often should kidney function and imaging be monitored?,Are there any dietary changes or lifestyle modifications we should make?,What are the chances that future children could have the same condition?,Are there any clinical trials or new treatments being studied for this condition?

Common questions about Genetic non-syndromic renal or urinary tract malformation

What is Genetic non-syndromic renal or urinary tract malformation?

Genetic non-syndromic renal or urinary tract malformation refers to a group of birth defects affecting the kidneys, ureters, bladder, or urethra that are caused by changes in a person's genes. The term 'non-syndromic' means these malformations occur on their own, without being part of a larger syndrome that affects other body systems. These malformations are also known as congenital anomalies of the kidney and urinary tract (CAKUT) when they have a genetic basis and appear in isolation. The types of malformations can vary widely. They may include kidneys that did not form properly (renal agen

At what age does Genetic non-syndromic renal or urinary tract malformation typically begin?

Typical onset of Genetic non-syndromic renal or urinary tract malformation is neonatal. Age of onset can vary across affected individuals.