Last-Hope drug access opens for devastating rare diseases
NCT ID NCT05222412
Summary
This program provides access to the investigational drug iptacopan for patients with two rare, serious conditions: C3 glomerulopathy (a kidney disease) and paroxysmal nocturnal hemoglobinuria (a blood disorder). It's for patients who have no other treatment options available and cannot join clinical trials. The goal is to control these diseases in people with serious or life-threatening cases when standard treatments have failed.
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Contacts and locations
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Conditions
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