News

Novartis has shared positive results from a phase 3b study of Fabhalta (iptacopan) in a new population of patients with the rare blood disorder paroxysmal nocturnal haemoglobinuria (PNH). The ...
Preservation of PNH Cells: Continuous lysis of Red Blood Cells in PNH leads to a dramatic reduction in total RBC count, often exceeding 80 to 90%.
Figure. NovelMed Scientist Prepares Complement Test Assays to Support Clinical Development of Ruxoprubart (NM8074) for Paroxysmal Nocturnal Hemoglobinuria (PNH). Ruxoprubart works by binding to Bb and ...
These interim data demonstrate that Ruxoprubart, administered as monotherapy, was safe, well-tolerated, and met all primary efficacy endpoints. Ruxoprubart achieved transfusion avoidance, increased ...
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disease that affects red blood cells, causing symptoms like dark urine, fatigue, and unexplained bruising. Early diagnosis is challenging due to ...
PNH treatments are better than ever before, and there’s hope they’ll continue to improve. Find out what’s in the works and how you can help.
If you have PNH, you will find that your condition requires constant management. But it’s important to be able to confront it head-on.
However, when researchers adjusted for PNH’s rarity (3.81 cases per 100,000 individuals), the PPV decreased to 19.59% (95% CI, 7.63% to 41.81%), suggesting that approximately 1 in 5 flagged ...
Alexion, AstraZeneca Rare Disease, announced that the National Institute for Health and Care Excellence (NICE) has given a positive recommendation for Voydeya (danicopan). This first-in-class, oral ...
Novartis’ Fabhalta (iptacopan) has been recommended by the National Institute for Health and Care Excellence (NICE) to treat paroxysmal nocturnal haemoglobinuria (PNH) in adults with haemolytic ...
Session ID: 2025-05-09:e6fca51e977014ed2969079 Player Element ID: vjs_video_3 ...
The Food and Drug Administration (FDA) has granted Orphan Drug designation to NM5072 for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). NM5072 is an investigational monoclonal ...