Article
Paroxysmal nocturnal hemoglobinuria from bench to bedside.
Clinical and translational science - 1 Jun 2011
Pu Jeffrey J, Brodsky Robert A
Abstract excerpt
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematologic disease that presents with protean manifestations. Clinical and laboratory investigation over the past 25 years has uncovered most of the basic science underpinnings of PNH and has led to the development of a highly effective targeted therapy. PNH originates from a multipotent hematopoietic stem cell (HSC) that acquires a somatic mutation in a gene...
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