by Peter Ciszewski | Oct 10, 2018
The FDA approved Revcovi (elapegademase-lvlr) injection in the United States, for the treatment of adenosine deaminase severe combined immune deficiency (ADA-SCID) in pediatric and adult patients. “We are gratified by the FDA’s timely recognition of...
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...
