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In this exclusive interview during WORLDSymposium 2015, Nicholas Clayton, MSc, staff scientist at Genzyme Corporation discusses the exciting new data he and his colleagues presented on antisense therapy that is in development to treat patients with Pompe disease.
Dr. Marc Patterson a pediatric neurologist at Mayo Clinic discusses Niemann-Pick disease type C.
Priya Kishnani, MD, professor of Pediatrics at Duke University School of Medicine and practicing geneticist at Duke Children's Hospital discusses our current understanding of the pathophysiology and management of Pompe disease.