Long-term treatment with Empaveli (pegcetacoplan) was generally well tolerated among adults with paroxysmal nocturnal hemoglobinuria (PNH), with the rate of injection-site reactions declining over time. That’s according to data from the Phase 3 PEGASUS clinical trial (NCT03500549) and the open-label extension (OLE) Study 307 (NCT03531255) that spanned…
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,…
From signing up for a patient panel webinar to swapping out social media profile photos, participants in PNH Awareness Week — observed this year Sep. 3-10 — are doing their part to call attention to paroxysmal nocturnal hemoglobinuria (PNH), a very rare acquired disease with which some 500 U.S.
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,”…
Unusual colon lesions caused by ischemic colitis led to the diagnosis of paroxysmal nocturnal hemoglobinuria (PNH) in an 82-year-old woman in Japan, but appropriate treatment eased her symptoms. According to the authors, her case highlights the importance of considering underlying diseases when unusual features are observed in a person…
Treatment with OMS906 is showing an ability to normalize all markers of red blood cell destruction, or hemolysis, in severely anemic people with paroxysmal nocturnal hemoglobinuria (PNH) never given a complement inhibitor. According to a five-month interim analysis of a Phase 1b proof-of-concept study (NCT05889299), once monthly, under-the-skin…
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…
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