by Peter Ciszewski | Apr 21, 2021
Miganush Stepanians, PhD, President and CEO of PROMETRIKA, a clinical research organization (CRO), discusses study designs used in rare disease clinical trials and the common struggles researchers face when designing and conducting these trials. Generally, the gold...
by Peter Ciszewski | Apr 19, 2021
Deborah Marsden, MD, Global Medical Expert, Medical Affairs, Ultragenyx, discusses the potential connection between mucopolysaccharidosis type VII (MPS VII) survival and non-immune hydrops fetalis (NIHF). MPS VII, or Sly syndrome, is a rare progressive...
by Peter Ciszewski | Apr 19, 2021
Owen A. O’Connor, Chief Scientific Officer at TG Therapeutics, gives an overview of follicular lymphoma (FL) and marginal zone lymphoma (MZL). FL is typically an indolent form of non-Hodgkin lymphoma (NHL) that arises from B-lymphocytes. It is the second...
by Peter Ciszewski | Apr 16, 2021
Cedric Francois, MD, PhD, Co-Founder & CEO of Apellis Pharmaceuticals, gives an overview of paroxysmal nocturnal hemoglobinuria (PNH). As Dr. Francois explains, PNH is a rare genetic blood disorder characterized by hemolytic anemia, thrombosis, and...
by Peter Ciszewski | Apr 16, 2021
Dalia Moawad, MD, Executive Director, Head of Neurological Rare Diseases, Medical Affairs at Genentech, outlines the company’s Evrysdi (risdiplam) clinical development program. This program was designed to represent a broad spectrum of people living with spinal...
