by Peter Ciszewski | Oct 5, 2018
The FDA expanded the approval of Hemlibra (emicizumab-kxwh) to include routine prophylaxis to prevent or reduce the frequency of bleeding episodes among individuals with hemophilia A without factor VIII inhibitors. Hemlibra is now the only prophylactic treatment for...
by Peter Ciszewski | Oct 5, 2018
Philip John Brooks, PhD, Program Director, Office of Rare Diseases Research, National Center for Advancing Translational Sciences, National Institutes of Health (NIH) discusses his work in rare diseases at the NIH. Dr. Brooks is focused on accelerating clinical...
by Peter Ciszewski | Oct 4, 2018
Katherine Stueland, Chief Commercial Officer at Invitae, discusses the role of genetic companies in assisting patients and physicians in diagnosing rare diseases. Katherine also highlights the important relationship between physicians, patients, and genetic...
by Peter Ciszewski | Oct 4, 2018
Sarepta Therapeutics announced that at the 23rd International Congress of the World Muscle Society in Mendoza, Argentina, Jerry Mendell, MD, of Nationwide Children’s Hospital presented positive updated results from its gene therapy clinical trial assessing...
by Peter Ciszewski | Oct 3, 2018
Greg Duncan, CEO of Celtaxsys discusses cystic fibrosis (CF) and the role of pulmonary exacerbations. Celtaxsys recently announced top line results of its Phase 2 EMPIRE-CF trial evaluating oral, once daily anti-inflammatory molecule, acebilustat, for the...
