by Peter Ciszewski | Apr 15, 2021
Owen A. O’Connor, Chief Scientific Officer at TG Therapeutics, discusses umbralisib, which was approved for the treatment of relapsed or refractory follicular lymphoma (FL) and relapsed or refractory marginal zone lymphoma (MZL). In the first part of the...
by Peter Ciszewski | Apr 14, 2021
Cedric Francois, MD, PhD, Co-Founder & CEO of Apellis Pharmaceuticals, discusses the results of the PEGASUS study evaluating the efficacy and safety of pegcetacoplan in patients with paroxysmal nocturnal hemoglobinuria (PNH). PNH is a rare, life-threatening...
by Peter Ciszewski | Apr 14, 2021
Jennifer Ibrahim, MD, Head of North America Medical Affairs, Rare Disease at Sanofi Genzyme, explains the difference between a geneticist and a genetic counselor. According to Dr. Ibrahim, medical geneticists are physicians trained in the identification and...
by Peter Ciszewski | Apr 14, 2021
David Dale, MD from the University of Washington and Sarah Cohen, MD, Medical Director, Rare Diseases at X4 Pharmaceuticals discuss the results from the Phase 2 dose-escalation and long-term extension study of mavorixafor in WHIM syndrome. WHIM syndrome is a...
by Peter Ciszewski | Apr 13, 2021
Jean Donadieu, MD, French Severe Chronic Neutropenia Registry; David Dale, MD, University of Washington, Seattle; Jolan Walters, MD, PhD at the University of South Florida at Johns Hopkins All Children’s Hospital; and Raffaele Badolato, MD, PhD at the...
