Paula Ragan, CEO and President, X4 Pharmaceuticals 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.
#WHIMSyndrome #WHIM #RareGeneticDiseases #mavorixafor #CXCRA #Waldenstrom
X4Pharma.com
Download the transcript here
Empowered Patient Podcast