by Peter Ciszewski | Jul 2, 2019
Durhane Wong-Rieger is the president and CEO of the Canadian Organization of Rare Disorders (CORD). CORD is a non-profit organization that represents over 100 patient organizations in educating Canadian policy makers on the value of Canada developing a Rare...
by Peter Ciszewski | Jul 1, 2019
At the BIO International Convention recently held in Philadelphia, PA, we talked with Christina Hartman, Senior Director of Advocacy & Policy for the EveryLife Foundation for Rare Diseases about the origins of this amazing organization. Hartman said, “The...
by Peter Ciszewski | Jul 1, 2019
Stephen Agyenim-Boateng, PharmD, RPH, is a medical consultant and board member of the non-profit organization, Sickle Cell 101. Sickle cell disease is often thought of as mostly afflicting people of African or African-American descent. That is inaccurate. The...
by Peter Ciszewski | Jun 28, 2019
The Food and Drug Administration (FDA) granted orphan drug designation to LM-030, an investigational therapy licensed from Novartis and ready to enter into pivotal clinical trials for the treatment of Netherton Syndrome. Netherton Syndrome is a severe autosomal...
by Peter Ciszewski | Jun 27, 2019
Akshay Vaishnaw, MD, PhD, President, Research and Development at Alnylam Pharmaceuticals, a company focused on RNA interference (RNAi) to develop new therapeutics for genetic conditions. At the BIO International Convention held in Philadelphia, PA, we talked to...
