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...
by Peter Ciszewski | Jun 26, 2019
John Maslowski is President and Chief Executive Officer (CEO) of Fibrocell Science, a biotech company that is developing cell and gene therapies for a variety of rare, mostly skin-related, conditions. At the BIO International Convention held in Philadelphia,...
