Mild to moderate thrombocytopenia is common in patients with PNH
(21) and the need for transfusion requirement varies widely (20).
Prior to these approvals, there were no therapies specifically available for the treatment of PNH
The FLAER test has been shown to permit a more accurate determination of the size of the PNH
clone as compared to standard flow cytometry.
is an acquired genetic blood disorder defined by hemolysis, in which patients' red blood cells are destroyed by complement, a component of the body's immune system.
It has been granted Orphan Drug Status in the PNH
indication from both the FDA and European regulatory agencies.
8220;We are once again very proud to be recognized and mentioned on the OCBJ's prestigious annual list,” said Larry Weng, PNH
Technology Chief Executive Officer.
Complement protein C3 is the master regulator of complement protein C5 that mediates cell damage in PNH
Securing our proprietary intellectual property position in Japan now provides us an important foothold for commercialization of Soliris for PNH
Apellis' drug candidate APL-2, an inhibitor of complement component C3, is designed to provide PNH
patients with an alternative to the current standard-of-care.
This year PNH
Technology was able to provide a happy Thanksgiving for many in need.
Patients with PNH
lack naturally occurring proteins that ordinarily prevent terminal complement from causing the red blood cell destruction (hemolysis) that is central to the serious morbidities and mortality associated with PNH
In the study, (1) Soliris was associated with significant long-term clinical improvements in patients with PNH
, regardless of baseline degree of hemolysis, anemia or transfusion requirements.