Researchers determined patients with paroxysmal hemoglobinuria may benefit from receiving ravulizumab over the long term.

Diagnosing PNH can be hard because it is a rare disease with symptoms that overlap with other bone marrow disorders. For this reason, the process will involve several types of tests. A flow ...

Mosaicism arising as a result of a compensatory mutation in hematopoietic cells occurs in some individuals with FA. What are the effects? These investigators found that roughly 1 in 3 patients ...

discusses the selected treatment and the results of the case: So, what happened to this patient? Well, we started her on a C5 inhibitor because she had a very large PNH clone. You can see on the ...

2 While complement 5 (C5) inhibitors such as Soliris and Ultomiris have long been the foundation of treatment, the PNH landscape is evolving rapidly, with new therapies and emerging biosimilars ...

Ultomiris is a complement C5 inhibitor that has become a mainstay of PNH treatment, helping it become a $2.8 billion product in the first nine months of 2024. It offers reduced dosing frequency ...