ATYPICAL HEMOLYTIC UREMIC SYNDROME

This study is for adults with a rare blood disorder called aHUS who are currently receiving antibody infusions. Researchers want to see if switching to a daily pill called iptacopan is safe and works just as well to prevent dangerous blood clots. About 50 participants will take i…

This study looks at the long-term safety and how well the drug iptacopan works for people with a rare blood disorder called atypical hemolytic uremic syndrome (aHUS). About 65 adults who already finished a previous iptacopan study and are still benefiting from the drug will conti…

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 people with atypical hemolytic uremic syndrome (aHUS), a rare disease that damages red blood cells and kidneys. Researchers want to understand how certain rare gene mutations affect the disease and whether they can find drugs that fix these problems. Participa…

This study is testing a new method called RNA-Seq to find genetic causes of rare diseases in people who have not been diagnosed with standard genetic tests. Researchers will take skin samples from about 105 participants, including healthy volunteers and patients with known or sus…

This study looks back at medical records of 200 people diagnosed with atypical hemolytic uremic syndrome (aHUS) in Egyptian hospitals between 2010 and 2023. aHUS is a rare, life-threatening disease that damages kidneys and other organs. The goal is to describe patient characteris…

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…