Hereditary hemolytic uremic syndrome

This study will follow about 80 Polish patients with atypical hemolytic uremic syndrome (aHUS) who are receiving ravulizumab (Ultomiris) as part of routine care. Researchers will collect data from medical records and follow patients over time to see how well the drug controls the…

This study looks at the long-term safety and effectiveness of the drug iptacopan in people with a rare blood disorder called atypical hemolytic uremic syndrome (aHUS). Participants must have completed a previous iptacopan study and still benefit from the drug. The goal is to moni…

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

This study is testing a new blood test (the HMEC test) to see if it can better identify which patients with thrombotic microangiopathy (TMA) have a specific immune system problem driving their disease. TMA is a severe condition that damages kidneys and the brain. The researchers …

This study follows 75 pregnant women who have taken Ultomiris (ravulizumab) for conditions like PNH or aHUS. Researchers will track pregnancy outcomes and baby health up to one year after birth. The goal is to gather safety information, not to test a new treatment.

This study aims to improve diagnosis for people with rare genetic diseases whose standard genetic tests came back negative. Researchers will take a small skin sample and analyze RNA to find hidden genetic changes. The study involves 105 participants, including healthy volunteers …

This study looks at how blood cells interact and what goes wrong in inherited bleeding disorders like Glanzmann thrombasthenia. Researchers will analyze blood samples from up to 60 healthy volunteers and patients to find genetic defects. The goal is to better understand these con…

This study looks at people with atypical hemolytic uremic syndrome (aHUS), a rare disease that damages small blood vessels and kidneys. Researchers want to understand how certain rare gene mutations cause the disease by growing cells from blood samples in the lab. The goal is to …

This observational study follows 3,000 people with atypical hemolytic-uremic syndrome (aHUS), a rare and serious kidney condition. Researchers will collect safety and effectiveness data on patients treated with eculizumab or ravulizumab, as well as those receiving other care. The…