—A pilot study of 9 patients with aplastic anemia and paroxysmal nocturnal hemoglobinuria asked them to track their symptoms over a 6-month period. The results were encouraging, suggesting a place for ...

SÃO PAULO--(BUSINESS WIRE)--Pint Pharma and OrphanDC announced on July 25th that ANVISA, the National Health Surveillance Agency, approved, on July 25th, 2023, the drug EMPAVELI® (pegcetacoplan, ...

—These findings suggest that clone size at PNH onset may serve as a prognostic factor for disease burden and MAVEs/TEs, aiding in clinical decision-making about treatment initiation or maintenance.

Iptacopan demonstrated significant hemoglobin improvements in PNH patients transitioning from anti-C5 therapies, validating its efficacy as an oral monotherapy. The safety profile of iptacopan was ...

(RTTNews) - Swiss drug major Novartis AG (NVS) announced Friday that Fabhalta (iptacopan) has received a positive opinion from the Committee for Medicinal Products for Human Use or CHMP of the ...

Diagnosed at a young age with a rare and potentially life-threatening disease, one man learned the power of taking control. When South Carolinian Roger was diagnosed with paroxysmal nocturnal ...

Call your healthcare provider or get emergency medical care right away if you have any of these signs and symptoms of a serious infection: fever with or without chills, fever and a rash, fever with ...

Please provide your email address to receive an email when new articles are posted on . Ravulizumab led to maintained control of intravascular hemolysis and sustained stable hemoglobin levels ...

"An efficacious oral treatment with a demonstrated safety profile could be practice-changing for physicians and help relieve burdens experienced by people with PNH," said Vinod Pullarkat, MD, MRCP, ...

APPLY-PNH extension data show that continuous Fabhalta ® (iptacopan) treatment in adults with paroxysmal nocturnal hemoglobinuria (PNH) enabled sustained hemoglobin-level increases to near-normal (≥12 ...