New hope for rare blood disease: expanded access to combo therapy
NCT ID NCT06028594
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This program offers continued access to a combination of two drugs, pozelimab and cemdisiran, for people with paroxysmal nocturnal hemoglobinuria (PNH), a rare condition where red blood cells break apart. It is for patients who have already completed a related study and aims to monitor long-term safety. The treatment helps control the disease but does not cure it.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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