by Peter Ciszewski | Oct 8, 2019
Aquestive Therapeutics is a specialty pharmacy company that is highly focused on providing treatment options for persons unable to properly swallow medicine. In this video, Daniel Barber is Chief Operating Officer at Aquestive describes the three...
by Peter Ciszewski | Oct 7, 2019
David Anderson, PhD, CNS Research Lead at Spark Therapeutics talks about the numerous neurological conditions that have been identified and the concern that most of them are rare diseases and most of them are not being properly managed. “There is something like...
by Peter Ciszewski | Oct 4, 2019
Nick Kenny, PhD, Chief Scientific Officer at Syneos Health talks about the challenges in developing clinical trials for rare conditions. Dr. Kenny said there are a myriad of challenges. First among them is the limited number of patients that can qualify for a...
by Peter Ciszewski | Oct 3, 2019
Julie Raskin, Executive Director of Congenital Hyperinsulinism International (CHI) explains the three key issues the CHI provides to its community: 1. Support 2. Awareness 3. Research Regarding support, Raskin noted that CHI supports families in many ways,...
by Peter Ciszewski | Oct 2, 2019
The Orphan Drug Act was first signed into law in 1983. The Act provided a variety of incentives for pharmaceutical companies to develop medications specifically for rare diseases, including tax breaks and seven years of market exclusivity. In 1983, there were...