A rare, genetic thrombotic microangiopathy due to dysregulation of the alternative complement pathway and characterized by the triad of hemolytic anemia, thrombocytopenia, and acute renal dysfunction.
Comprehensive, easy-to-understand information about this condition
How we create this content →Research studies investigating treatments and therapies for this condition.
Active Trials
Total Trials
Data from ClinicalTrials.gov Jan 31, 2026
Research studies investigating treatments and therapies for this condition.
Active Trials
Total Trials
Data from ClinicalTrials.gov Jan 31, 2026
The limited documentation surrounding atypical hemolytic-uremic syndrome is due to its rarity, affecting fewer than 10 in 100,000 individuals. This extreme rarity limits the ability to conduct large-scale clinical studies, resulting in a lack of systematic documentation of clinical features and genetic underpinnings. As research continues, there is hope for a better understanding of this condition.
To navigate atypical hemolytic-uremic syndrome, seek a nephrologist with expertise in thrombotic microangiopathies. Consider reaching out to specialized centers that focus on rare kidney diseases. While there are currently no identified patient organizations, you can find support and resources through GARD at rarediseases.info.nih.gov. Additionally, inquire about participation in clinical trials or natural history studies that may provide further insights into your condition.
There are currently 17 active clinical trials investigating various aspects of atypical hemolytic-uremic syndrome. Two orphan drugs, eculizumab and ravulizumab-cwvz, have received FDA approval for treatment. For more information on ongoing trials, you can visit ClinicalTrials.gov at https://clinicaltrials.gov/search?cond=atypical%20hemolytic-uremic%20syndrome. This research landscape offers hope for improved management and treatment options.
Actionable guidance for navigating care for atypical hemolytic-uremic syndrome
To navigate atypical hemolytic-uremic syndrome, seek a nephrologist with expertise in thrombotic microangiopathies. Consider reaching out to specialized centers that focus on rare kidney diseases. While there are currently no identified patient organizations, you can find support and resources through GARD at rarediseases.info.nih.gov. Additionally, inquire about participation in clinical trials or natural history studies that may provide further insights into your condition.
Consider asking your healthcare providers these condition-specific questions
Helpful links for rare disease information and support
Clinical profile data for this condition is not yet available. Phenotype information may still be loading below.
Research studies investigating treatments and therapies for this condition.
Active Trials
Total Trials
Data from ClinicalTrials.gov Jan 31, 2026
Consider asking your healthcare providers these condition-specific questions
European rare disease database
Genetic and Rare Diseases Info Center
AI-Generated Content: This summary was generated using AI. Content has been fact-checked. Always consult with qualified healthcare providers for medical guidance.
Organizations with approved therapies for this disease
No approved therapies yet
Research is ongoing — 6 companies have orphan drug designations for this disease
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