Overview
Nephrotic syndrome without extrarenal manifestations is a kidney disorder where the tiny filtering units in the kidneys, called glomeruli, become damaged and leak large amounts of protein into the urine. Unlike some forms of nephrotic syndrome that affect other organs, this condition is limited to the kidneys. The name means that the disease does not cause problems outside the kidneys (no 'extrarenal' or 'beyond the kidney' effects). When the kidneys leak protein, the body loses important proteins from the blood. This leads to a buildup of fluid in the body, causing swelling — especially around the eyes, legs, and belly. Low protein levels in the blood also make it harder for the body to fight infections and can raise the risk of blood clots. Common symptoms include puffy eyes in the morning, swollen ankles and feet, foamy or frothy urine, weight gain from fluid, and fatigue. Treatment usually focuses on reducing protein loss, controlling swelling, and protecting the kidneys from further damage. Doctors often use steroid medicines as a first step. Some patients respond well to steroids, while others need additional medications. In cases linked to a specific genetic change, treatment may be tailored to the underlying cause. Regular monitoring by a kidney specialist (nephrologist) is essential to manage this condition and preserve kidney function over time.
Key symptoms:
Swelling around the eyes, especially in the morningSwollen ankles, feet, and legsSwollen belly from fluid buildupFoamy or frothy urineUnexplained weight gain from fluid retentionFatigue and low energyReduced amount of urineLoss of appetitePale, dull skinIncreased risk of infectionsHigh cholesterol levels in the bloodBlood clots in some cases
Variable
Can be inherited in different ways depending on the underlying gene
Variable
Can begin at different ages, from infancy through adulthood
FDA & Trial Timeline
9 eventsBrigham and Women's Hospital — PHASE1
Mario Negri Institute for Pharmacological Research — PHASE2
Consun Pharmaceutical Group — PHASE1
Travere Therapeutics, Inc. — PHASE4
Gødstrup Hospital — NA
Military Institute od Medicine National Research Institute — PHASE3
Travere Therapeutics, Inc. — PHASE2
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
No FDA-approved treatments are currently listed for Nephrotic syndrome without extrarenal manifestations.
7 clinical trialsare actively recruiting — trials can provide access to cutting-edge therapies.
View clinical trials →Specialists
View all specialists →No specialists are currently listed for Nephrotic syndrome without extrarenal manifestations.
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
Travel Grants
No travel grants are currently matched to Nephrotic syndrome without extrarenal manifestations.
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Caregiver Resources
NORD Caregiver Resources
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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 is the most likely cause of my nephrotic syndrome, and should I have genetic testing?,How will we know if my treatment is working, and what are the signs of a relapse?,What are the long-term risks to my kidney function, and how can we slow any damage?,Are there clinical trials or newer treatments I should consider?,What dietary changes do I need to make, and should I see a dietitian?,What vaccinations do I need, and are there any I should avoid while on immunosuppressive treatment?,At what point would a kidney transplant be considered, and how does that process work?
Common questions about Nephrotic syndrome without extrarenal manifestations
What is Nephrotic syndrome without extrarenal manifestations?
Nephrotic syndrome without extrarenal manifestations is a kidney disorder where the tiny filtering units in the kidneys, called glomeruli, become damaged and leak large amounts of protein into the urine. Unlike some forms of nephrotic syndrome that affect other organs, this condition is limited to the kidneys. The name means that the disease does not cause problems outside the kidneys (no 'extrarenal' or 'beyond the kidney' effects). When the kidneys leak protein, the body loses important proteins from the blood. This leads to a buildup of fluid in the body, causing swelling — especially arou
Are there clinical trials for Nephrotic syndrome without extrarenal manifestations?
Yes — 7 recruiting clinical trials are currently listed for Nephrotic syndrome without extrarenal manifestations on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.