Niemann-Pick Disease
TRND Research Leads to NIH Trial to Test Drug for Niemann-Pick Type C1
Today, a promising new treatment is on the horizon. On Jan. 23, 2013, NIH initiated a Phase I clinical trial to evaluate the safety and effectiveness of cyclodextrin as a potential therapy for Niemann-Pick type C1.
This progress is the result of the collaborative efforts of an award-winning, multidisciplinary team of experts from NCATS, the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), the National Institute of Neurological Disorders and Stroke (NINDS), and the National Human Genome Research Institute; Janssen Research & Development, LLC; Washington University in St. Louis School of Medicine; Albert Einstein School of Medicine, New York City; and University of Pennsylvania, Philadelphia.