The gene mutation that causes PNH occurs in several steps. First, a mutation develops in the phosphatidylinositol glycan class A (PIGA) gene, which helps create proteins that protect blood cells ...

With biosimilars to AstraZeneca’s blockbuster rare blood disease med Soliris recently available in the U.S., nonprofit health ...

PiaSky (crovalimab) is a complement C5 inhibitor, a class recognised as the standard ... as patients with PNH require lifelong therapy. PiaSky's approval by the MHRA earlier this year was based ...

The first-in-class therapy – a targeted factor B inhibitor – was able to "substantially" reduce the destruction of red blood cells (haemolysis) that occurs in PNH when used over 12 weeks as a ...