Joe Pugliese of the Hemophilia Alliance, a not-for-profit organization of federally funded hemophilia treatment centers that is working to ensure its member have the expertise, resources and public support to sustain their integrated clinical and pharmacy services for persons with hemophilia and other bleeding disorders.
The U.S. Food and Drug Administration (FDA) granted approval to Scenesse (afamelanotide) to increase pain-free light exposure in adult patients with a history of phototoxic reactions (damage to skin) from erythropoietic protoporphyria (EPP).
Nick Kenny, PhD, Chief Scientific Officer at Syneos Health talks about the challenges in developing clinical trials for rare conditions.
Julie Raskin, Executive Director of Congenital Hyperinsulinism International (CHI) explains the three key issues the CHI provides to its community.
Badhiu Basu, PhD, owner of the Converge Advisory Group talked about the Orphan Drug Act and what it has (and has not) accomplished.