by Peter Ciszewski | May 15, 2020
Ozlem Goker-Alpan, MD, provides a summary of research presented at WORLDSymposium 2020 focused on Fabry disease. This video resource is part of the CME activity Fabry Disease Highlights from WORLDSymposium 2020, which is supported by an educational grant from...
by Peter Ciszewski | May 14, 2020
Rami Levin, CEO of Saniona, a pharmaceutical company focused on rare neurological diseases describes Prader-Willi syndrome (PWS) and the latest clinical data involving PWS patients given Tesomet (tesofensine + metoprolol). PWS is a genetic condition that leads...
by Peter Ciszewski | May 13, 2020
Michael Snape, PhD, Chief Scientific Officer at AMO Pharma describes the history of his company and its current pipeline that is largely focused on rare neurodevelopmental conditions, including congenital myotonic dystrophy, Phelan-McDermid syndrome, and Rett...
by Peter Ciszewski | May 12, 2020
Riam Shammaa, MD, Founder INTELLiSTEM Technologies, lists three factors that are important to understand when developing a vaccine for COVID-19. The three factors mentioned by Dr. Shammaa are 1) understand that the virus is mutating, 2) have a cellular response...
by Peter Ciszewski | May 11, 2020
Shannon Palmatier has two sons with progressive familial intrahepatic cholestasis (PFIC), a rare hereditary disease in which children are unable to properly remove bile acids from their liver. The disease is a genetic disorder that follows an autosomal...
