FDA Approves Ruxolitinib to Treat Chronic GVHD

The Food and Drug Administration (FDA) has approved ruxolitinib (Jakafi)  to treat chronic graft-versus-host disease (GVHD) after failure of one or two lines of systemic therapy in patients 12 years and older. Chronic GVHD is a rare complication that can occur after a...

When to Suspect AADC Deficiency in a Baby

  Kylie O’Keefe, Senior Vice President, Head of Global Commercial and Corporate Strategy at PTC Therapeutics, describes early signs and symptoms of aromatic L-amino acid decarboxylase (AADC) deficiency. AADC deficiency is a rare, genetic disorder due to a defect...

 Targeted Therapy for Huntington Disease

  Michael Panzara, MD, MPH, Chief Medical Officer at Wave Life Sciences, provides an overview of the pathophysiology of Huntington disease and how that knowledge is guiding treatment development. Huntington disease is an inherited, neurodegenerative disorder that...