by Peter Ciszewski | Jul 29, 2018
Heather A. Lau, MD, Director, Lysosomal Storage Disease Program at NYU Langone in New York City discusses Fabry disease, an inherited disorder that results from the buildup of a particular type of fat, called globotriaosylceramide, in the body’s cells....
by Peter Ciszewski | Jul 28, 2018
Daniel de Boer, Founder and CEO of ProQR, discusses his company’s working relationships with various rare disease advocacy groups. ProQR has several programs in devleopment for rare diseases including several forms of inherited blindness such as Leber’s...
by Peter Ciszewski | Jul 27, 2018
Gail Cawkwell, MD, PhD, Senior Vice President, Medical Affairs at Intercept, discusses current research in primary biliary cholangitis (PBC), a rare liver disease that is caused by an autoimmune reaction. When bile is not able to move through the bile ducts, it...
by Peter Ciszewski | Jul 26, 2018
Editor’s Note: This interview was conducted at #Bio2018. Please pardon the background noise. Barry Greene, President of Alnylam, provides an overview of his company and the science of RNA interference (RNAi). Alnylam is developing RNAi (RNA interference)...
by Peter Ciszewski | Jul 25, 2018
Mr. Robert (Bob) E. Ward, Chairman of the Board and Chief Executive Officer of Eloxx Pharmaceuticals discusses the success of the Orphan Drug Act (ODA). This Act was first enacted in the United States in 1983 and set up to encourage the development of drugs for...
