ATYPICAL HEMOLYTIC UREMIC SYNDROME

This study looks at the long-term safety and effectiveness of the drug iptacopan in people with atypical hemolytic uremic syndrome (aHUS), a rare and serious kidney condition. About 65 adults who already completed a previous iptacopan study and are benefiting from the drug will c…

This study is testing a drug called ravulizumab in people with atypical hemolytic uremic syndrome (aHUS), a rare condition that causes blood clots and kidney damage. The main goal is to see if the drug improves platelet counts over 26 weeks. About 20 participants will receive the…

This study looks at whether adults with aHUS (a rare blood disorder) can safely switch from their current infusion treatment to a daily pill called iptacopan. About 50 participants will take iptacopan for 12 months to see if it prevents dangerous blood clots. The goal is to find …

This study looks at people with atypical hemolytic uremic syndrome (aHUS), a rare kidney disease. Researchers want to understand how certain rare gene mutations affect the disease. They will grow cells from patients and healthy donors in the lab to study them. The goal is to find…

This study looks back at the medical records of 200 people in Egypt diagnosed with atypical hemolytic uremic syndrome (aHUS), a rare and serious disease that can damage the kidneys and other organs. Researchers want to learn more about the patients' characteristics, how the disea…

This study aims to improve diagnosis for people with rare genetic diseases that standard DNA tests cannot identify. Researchers will use RNA sequencing on skin cells from 105 participants, including healthy volunteers and patients with known or suspected genetic conditions. The g…

This study is a large, long-term registry that follows up to 3,000 people with atypical hemolytic-uremic syndrome (aHUS), a rare blood disorder. Researchers will collect safety and health information from both treated and untreated patients to better understand the disease and it…