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),…
Monthly injections of the experimental therapy crovalimab controlled red blood cell loss and the need for blood transfusions for up to four years in adults with paroxysmal nocturnal hemoglobinuria (PNH), according to data from a long-term extension study. In fact, more than 80% — and up to 92% —…
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 single dose of Homology Medicines’ gene therapy candidate HMI-104 led to the sustained production of a therapeutic antibody to treat paroxysmal nocturnal hemoglobinuria (PNH), preclinical studies suggest. “Given the severity of PNH, which is a rare, acquired blood disorder, and the unmet need associated with chronically administered…
Iptacopan, an experimental oral medication being developed by Novartis, increased hemoglobin to near-normal levels in people with paroxysmal nocturnal hemoglobinuria (PNH) — without the need for blood transfusions — according to data from two global Phase 3 clinical trials. The trials, APPLY-PNH (NCT04558918) and APPOINT-PNH (NCT04820530),…
An experimental therapy being developed by Omeros can nearly double the level of hemoglobin safely after three doses in severely anemic people with paroxysmal nocturnal hemoglobinuria (PNH). That’s according to interim data from an ongoing Phase 1b clinical trial that’s evaluating the effect of once-monthly subcutaneous (under-the-skin) injections…
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