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Targeted Therapy for WHIM Syndrome Shows Promise
NIAID study is the first to examine long-term use of plerixafor in any disease
NIAID study is the first to examine long-term use of plerixafor in any disease
WHIM syndrome is a rare genetic disease in which patients lack sufficient infection-fighting white blood cells in their bloodstream. With white blood cells trapped in the bone marrow, patients experience frequent bacterial and viral infections, recurring skin and genital warts, and an increased risk of developing cancer caused by human papillomavirus.
Current standard therapy for WHIM syndrome does not target the genetic defect, is difficult to administer, and is not completely effective. NIAID scientists provide evidence that the drug plerixafor may be a safe and effective therapy. The study appears in the February 12, 2014, online issue of Blood.
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