NovelMed Therapeutics has launched a Phase 2 clinical trial to test its experimental therapy, NM8074, in people with paroxysmal nocturnal hemoglobinuria (PNH) who haven’t received treatment for the disease. Eight patients have been enrolled so far and two have started treatment, according to the company. The Phase…
A proof-of-concept clinical trial of the experimental oral therapy BCX10013 is enrolling people with paroxysmal nocturnal hemoglobinuria (PNH) in Cape Town, South Africa, with plans to expand to other sites where approved PNH treatments are not available. The Phase 1 study (NCT06100900) is open to adults with PNH…
The experimental treatment cemdisiran (ALN-CC5) failed to fully control hemolysis, or red blood cell destruction, in a small clinical trial involving people with paroxysmal nocturnal hemoglobinuria (PNH). However, the therapy’s use was found to reduce the dose of Soliris (eculizumab) needed to keep blood cell destruction under control…
The U.S. Food and Drug Administration (FDA) has agreed to review an application seeking the approval of the monthly subcutaneous (under-the-skin) injection therapy crovalimab as a treatment for paroxysmal nocturnal hemoglobinuria (PNH), according to its developer, Genentech. “This filing acceptance reinforces the value of crovalimab,” Levi Garraway, MD,…
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…
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