A European Medicines Agency committee has recommended that Novartis’ twice-daily oral therapy Fabhalta (iptacopan) be approved for the treatment…
Articles by Marisa Wexler MS
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to ruxoprubart, an experimental treatment for paroxysmal…
Danicopan has been approved in Japan as a first-in-class, add-on therapy for people with paroxysmal nocturnal hemoglobinuria (PNH) who…
NovelMed Therapeutics has launched a Phase 2 clinical trial to test its experimental therapy, NM8074, in people with…
A proof-of-concept clinical trial of the experimental oral therapy BCX10013 is enrolling people with paroxysmal nocturnal hemoglobinuria (PNH) in…
The experimental treatment cemdisiran (ALN-CC5) failed to fully control hemolysis, or red blood cell destruction, in a small clinical trial…
The U.S. Food and Drug Administration (FDA) has agreed to review an application seeking the approval of the monthly subcutaneous…
When used in real-world practice to treat paroxysmal nocturnal hemoglobinuria (PNH), the safety and effectiveness of Ultomiris (ravulizumab-cwvz)…
Treatment with the experimental medication zilucoplan reduced blood cell destruction in previously untreated people with paroxysmal nocturnal hemoglobinuria (PNH)…
A rare disease, paroxysmal nocturnal hemoglobinuria (PNH) affects roughly 1 in of every 237,000 people in Brazil, a study…