Marisa Wexler MS,  —

When used in real-world practice to treat paroxysmal nocturnal hemoglobinuria (PNH), the safety and effectiveness of Ultomiris (ravulizumab-cwvz) is similar to what’s been reported in clinical trials, according to a recent study from Japan. “These real-world data support the safety and effectiveness of [Ultomiris] treatment in Japanese patients,”…

Treatment with the experimental medication zilucoplan reduced blood cell destruction in previously untreated people with paroxysmal nocturnal hemoglobinuria (PNH) in Phase 2 clinical trials. However, PNH patients who switched to zilucoplan from an approved PNH therapy experienced more blood cell destruction, results showed. That’s according to findings detailed…

A rare disease, paroxysmal nocturnal hemoglobinuria (PNH) affects roughly 1 in of every 237,000 people in Brazil, a study estimates. Its findings shed light on common comorbidities, or co-occurring health conditions like aplastic anemia, affecting PNH patients in that country, as well as the causes of death in those…

The high cost of Soliris (eculizumab) is likely to have catastrophic consequences for people with paroxysmal nocturnal hemoglobinuria (PNH) in Nepal and other places with limited healthcare resources. That’s the argument a team led by scientists in Nepal made in the Orphanet Journal of Rare Diseases, in…

Treatment with crovalimab, an experimental under-the-skin injection therapy, effectively prevented red blood cell death, reduced the need for blood transfusions, and eased fatigue in adults and adolescents with paroxysmal nocturnal hemoglobinuria (PNH). That’s according to a primary analysis of data from the Phase 3 COMMODORE 3 trial (NCT04654468),…

Treatment with the experimental complement-blocking therapy omoprubart reduced markers of hemolysis, or blood cell destruction, in people with paroxysmal nocturnal hemoglobinuria (PNH). Those findings, from preliminary data of a Phase 1/2 study, were announced by its developer, China-based Canbridge Pharmaceuticals, which now is planning to launch a pivotal…

Chugai Pharmaceutical has applied to regulatory authorities in Japan seeking the approval of crovalimab, an under-the-skin injection therapy that can be administered at home, to treat paroxysmal nocturnal hemoglobinuria (PNH). “We are very pleased that crovalimab, our…

NM3086, an experimental therapy for paroxysmal nocturnal hemoglobinuria (PNH) that’s designed to block the alternative complement pathway’s activation, was generally well tolerated in a first in-human clinical trial. Findings from the Phase 1 study with healthy volunteers suggest NM3086 is working as intended, according to its developer, NovelMed.

Varicella-zoster virus — which causes chickenpox and shingles — can lead to fatal infections in people with paroxysmal nocturnal hemoglobinuria (PNH) who are on immune-suppressing treatment, a case report highlights. Researchers urge clinicians to be aware of signs of possible infection in a report, “Disseminated varicella-zoster…

A bone marrow transplant may be an effective treatment for some people with bone marrow cancer who develop subsequent or co-occurring paroxysmal nocturnal hemoglobinuria (PNH), a study indicates. The study, “Haemolytic paroxysmal nocturnal haemoglobinuria in patients with myeloid neoplasms: A rare association with specific therapeutic implications,”…