New hope for rare blood and kidney diseases: early access to iptacopan
NCT ID NCT05222412
First seen Jan 05, 2026 · Last updated May 14, 2026 · Updated 23 times
Summary
This program offers early access to the drug iptacopan for people with serious conditions like C3 glomerulopathy (a kidney disease) or paroxysmal nocturnal hemoglobinuria (a blood disorder) who have no other treatment options. Doctors can request the drug for patients who are not eligible for clinical trials or have exhausted all standard therapies. The goal is to provide a potential treatment when nothing else is available.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
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