by Peter Ciszewski | Sep 23, 2020
Emil Kakkis, MD, President and Founder of Ultragenyx Pharmaceutical Inc., explains how the Orphan Drug Act of 1983 may have been the catalyst to begin developing drugs for rare diseases, other factors are now in place to make orphan drug development a viable...
by Peter Ciszewski | Sep 22, 2020
Kealey-Shay Spolijarevic has progressive familial intrahepatic cholestasis (PFIC), a rare hereditary disease in which an individual is unable to properly remove bile acids from their liver. She describes a few of the treatment options available for PFIC...
by Peter Ciszewski | Sep 18, 2020
Stormy Chamberlain, PhD, from the University of Connecticut, discusses the recently announced research collaboration between UConn and Ovid Therapeutics to accelerate the development of a short hairpin RNA (shRNA)-based therapy for the treatment of Angelman...
by Peter Ciszewski | Sep 17, 2020
The U.S. Food and Drug Administration (FDA) has granted a Priority Review to assess arimoclomol to treat children with Niemann-Pick disease Type C. A Priority Review means that the FDA will review the drug within 6 months instead of the standard 10 months. A...
by Peter Ciszewski | Sep 15, 2020
Kealey-Shay Spolijarevic has progressive familial intrahepatic cholestasis (PFIC), a rare hereditary disease in which an individual is unable to properly remove bile acids from their liver. She gives a message to other PFIC patients that once you are...
