Sanfilippo Syndrome (MPS III) Overview

  Heather A. Lau, MD, Director, Lysosomal Storage Disease Program at NYU Langone in New York City discusses Mucopolysaccharidosis type III (MPS III), also known as Sanfilippo syndrome. This progressive disorder primarily affects the brain and spinal cord (central...

FDA Approves Romiplostim for Children With Immune Thrombocytopenia

The FDA approved romiplostim for treatment of pediatric patients with immune thrombocytopenia. The approval of romiplostim (Nplate, Amgen) applies to children aged 1 year or older who have had immune thrombocytopenia (ITP) for at least 6 months and had insufficient...

Vascular Ehlers-Danlos Syndrome (vEDS)

  William T. Andrews, MD, FACP, Chief Medical Officer of Acer Therapeutics discusses his company’s focus on Vascular Ehlers-Danlos Syndrome (vEDS). Vascular Ehlers-Danlos syndrome (previously known as EDS Type IV or EDS IV) is an inherited connective tissue...
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