Positiva resultat för nytt läkemedel mot ovanlig blodsjukdom

Läkemedlet pegcetacoplan förbättrade tillståndet hos patienter vid den allvarliga och svårbehandlade sjukdomen PNH, enligt en studie.

By Tobias Bergman

March 18, 2021 4:56 PM

Apellis Pharma och Sobi meddelade idag resultaten med läkemedlet pegcetacoplan i The New England Journal of Medicine. I en fas III-studie med 80 patienter gav läkemedlet bättre resultat än eculizumab vid behandling av den sällsynta sjukdomen Paroxysmal Nokturn Hemoglobinuri (PNH).

Paroxysmal Nokturn Hemoglobinuri är en ovanlig kronisk sjukdom som leder till låga nivåer av hemoglobin i röda blodkroppar vilket leder till att blodkropparna förstörs genom hemolys. Sjukdomen leder till trötthet, andningssvårigheter och kan kräva frekventa blodtransfusioner

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