by Peter Ciszewski | Mar 26, 2020
Philip M. Murphy, MD, Chief, Molecular Signaling Section, Laboratory of Molecular Immunology; Chief, Laboratory of Molecular Immunology, National Institute of Allergy and Infection, discusses the etiology and diagnosis of WHIM syndrome, a rare immunodeficiency...
by Peter Ciszewski | Mar 25, 2020
The U.S. Food and Drug Administration (FDA) has granted an Orphan Drug Designation to remdesivir to treat persons with Covid-19. Remdesivir is an antiviral agent currently in development by Gilead Sciences. The drug is not approved to treat patients with Covid-19 but...
by Peter Ciszewski | Mar 24, 2020
Petra Oliva, PhD of Archimed Life Science explains the value of testing for both Gaucher disease and Neimann-Pick disease in persons suspected of having one of those rare conditions. Gaucher disease is a rare lysosomal storage disorder due to a deficiency in...
by Peter Ciszewski | Mar 23, 2020
David Kronn, MD, Associate Professor of Pediatrics at New York Medical College, provides an overview of Pompe disease. Pompe disease is a lysosomal disorder that leads reduced levels of acid alpha-glucosidase (GAA). The net result is a buildup of glycogen in...
by Peter Ciszewski | Mar 20, 2020
The US Food and Drug Administration (FDA) has issued a guidance for industry, investigators, and institutional review boards in regard to how to conduct clinical trials during the current coronavirus (COVID-19) pandemic. The FDA recognizes that the current pandemic...
