Patients with paroxysmal nocturnal hemoglobinuria (PNH) in 53 countries with limited access to life-saving treatments will be able to receive treatment with Fabhalta (iptacopan). The Max Foundation, a global nonprofit organization, and Novartis are expanding their collaboration to address the unmet needs of patients across Sub-Saharan…
In rare cases, acute kidney injury may be sign of paroxysmal nocturnal hemoglobinuria (PNH), as described in a recent case report. “Although renal dysfunction in PNH is rare and as renal dysfunction is a dreadful complication, timely intervention is life-saving for the patient who requires hemodialysis,” the scientists wrote.
Treatment with Ultomiris (ravulizumab) enabled paroxysmal nocturnal hemoglobinuria (PNH) patients to achieve the same level of physical and mental health, daily activity, and sleep duration displayed by healthy individuals from the U.S. general population, according to the findings of an observational study. In the study, people with PNH…
A young woman with paroxysmal nocturnal hemoglobinuria (PNH) developed neutropenic enterocolitis (NEC), a severe and life-threatening complication, after receiving treatment with Soliris (eculizumab), a case report indicates. NEC is marked by severe inflammation of the large intestine, which may be associated with infection, due to a markedly low…
Increasing dosing of Empaveli (pegcetacoplan) followed by co-administering Soliris (eculizumab) effectively managed acute red blood cell destruction in a woman with paroxysmal nocturnal hemoglobinuria (PNH), a study shows. The effort underscores how acute red blood cell destruction “requires prompt diagnosis and treatment to avoid potentially…
A pregnant woman with paroxysmal nocturnal hemoglobinuria (PNH) and an inherited blood clotting disorder had a successful and uneventful pregnancy after receiving tailored treatment with Soliris (eculizumab) and anti-clotting therapy. “With the combined expertise of hematologists and obstetricians, comprehensive care can be provided to pregnant women with PNH, minimizing the…
Epysqli (SB12), a biosimilar of Soliris (eculizumab), is as effective as the reference therapy in reducing the need for blood transfusions in patients with paroxysmal nocturnal hemoglobinuria (PNH). That’s according to a post-hoc analysis of the pivotal Phase 3 SB12 study (NCT04058158), whose findings supported Epysqli’s approval in…
A rare case of portal vein thrombosis (PVT) — where a clot blocks blood flow in the portal vein, a major liver blood vessel — in a 49-year-old woman with paroxysmal nocturnal hemoglobinuria (PNH) was detailed in a case report. Transjugular intrahepatic portosystemic shunt (TIPS), a surgical procedure that…
Early mortality in people with paroxysmal nocturnal hemoglobinuria (PNH) is linked to being older and having heart or blood disorders and a history of solid tumors at diagnosis, a Danish study has found. The leading causes of death among patients were infections and associated blood-related diseases, while the approved…
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