https://lnkd.in/eMBKGUeZ Good news for the Paroxysmal nocturnal hemoglobinuria (PNH) patients. Though it is quite rate, I knew one patient with PNH when I was still treating patients. PNH is a rare immune blood disorder that causes red blood cells (RBC) to break down easily, leading to anemia, constant fatigue, and weakness. It also leads to blood clots and related complications due to issues related to platelets. Iptacopan is an oral drug, first in its class that selectively inhibits factor B, the active component of the complement’s C3 and C5 convertases (alternative complement pathway of the immune system) to control RBC destruction within and outside the blood vessels. The drug is approved with the appropriate name "Fabhalta". #PNH #Iptacopan #OralSelectiveFactorBinhbitor
