Addressing Genetic Mutation that Leads to Rare Disease of the Immune System with Paula Ragan X4 Pharmaceuticals TRANSCRIPT
Download PDFAug 25, 2020
Paula Ragan, CEO and President, X4 Pharmamceuticals is developing innovative approaches to treat patients suffering from rare diseases with an initial focus on WHIM syndrome, a rare disease of the immune system that has a basis in a genetic mutation. X4 is also focused on a rare form of lymphoma called Waldenstrom macroglobulinemia, a blood cancer also caused or driven by a gene mutation. The X4 drug mavorixafor is in capsule form and is a lifelong therapy that revs up the immune system by attaching to white blood cells.
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