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 ...

In the Phase IIIB APPULSE-PNH study, adult patients with paroxysmal nocturnal hemoglobinuria (PNH) who switched to Fabhalta experienced clinically meaningful improvements in hemoglobin (Hb) levels ...

Paroxysmal Nocturnal Hemoglobinuria (PNH) is a rare hematological (Blood) disorder.Regulatory approval for the Phase II trial in PNH subjects was granted based on the safety profile of healthy ...

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 b ...

Research has found limitations in current anti-C5 therapy, but there are also emerging complement-targeted treatments for PNH.

The machine learning model shows promise in detecting paroxysmal nocturnal hemoglobinuria (PNH) by assessing electronic health records (EHR) data, potentially transforming the diagnostic landscape ...

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 ...

NICE recommends Voydeya® (danicopan) with ravulizumab or eculizumab for treating paroxysmal nocturnal haemoglobinuria (PNH) in adults with residual haemolytic anaemia ...

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 ...

Dr. Haumschild drives a discussion providing an overview of PNH and pathology of the disease.