2024年11月5日 · In December 2023, iptacopan was approved as the first orally administered monotherapy for patients with PNH in the United States (US). Since then, two more CIs have …

2023年7月31日 · For instance, about 30 to 40% of PNH cases are reported in the United States(US) and Europe, whereas less than 10% of PNH cases are reported from Asia. …

2023年11月2日 · PNH is clinically heterogeneous, and hospitalization and regular blood transfusions may be needed. Treatment with currently approved C5 inhibitors (C5i), …

2019年11月13日 · The objective of this study was to estimate the incidence and prevalence of PNH and to describe real-world treatment patterns among patients newly diagnosed with PNH …

In the United States, researchers estimate the prevalence of monoclonal PNH cases at 3000 individuals and biclonal (or multiclonal) PNH cases at 2 individuals based on the 2010 US …

To date, the only therapies for PNH approved by the U.S. Food and Drug Administration (FDA) are the complement-inhibitory drugs eculizumab (approved in 2007) and ravulizumab …

Patients with paroxysmal nocturnal hemoglobinuria (PNH) often experience a lengthy path to diagnosis. Fewer than 40% of patients with PNH receive a diagnosis within 12 months of …

2024年4月15日 · NM5072 is being developed for PNH patients in the United States and globally. The FDA grants ODD status to new medication intended for the treatment, diagnosis or …

The objective of this study was to determine C5i treatment effects on clinical parameters, PNH symptoms, quality of life, and resource use for PNH patients. This cross-sectional study …

2024年11月5日 · This interim analysis aims to provide early insights into the characteristics and treatment patterns of patients enrolled in APPRISE-PNH to date. Methods: APPRISE-PNH is a …