by Peter Ciszewski | Nov 1, 2019
David Pearce, PhD, of the Coordination of Rare Diseases at Sanford (CoRDS) talks about their patient advocacy conference held in Sioux Falls, SD. The 3-day Patient Advocacy Certificate Training (PACT) is a program designed by CoRDs and Professional Patient...
by Peter Ciszewski | Nov 1, 2019
Daniel Barber is chief operating officer at Aquestive Therapeutics, a specialty pharmacy company that is highly focused on providing treatment options for persons unable to properly swallow medicine (e.g., people with epilepsy, ALS, Parkinson’s, etc). Recently,...
by Peter Ciszewski | Oct 31, 2019
The U.S. Food and Drug Administration (FDA) has accepted Genentech’s Biologics License Application (BLA) for satralizumab to treat adults and adolescents with neuromyelitis optica spectrum disorder (NMOSD). The FDA’s review will largely focus on two Phase III studies...
by Peter Ciszewski | Oct 30, 2019
Julie Raskin, Executive Director of Congenital Hyperinsulinism International (CHI) explains how congenital hyperinsulism is diagnosed and the latest research is underway to improve how it is diagnosed and understood. Congenital hyperinsulinism occurs in...
by Peter Ciszewski | Oct 29, 2019
Bobby Gasper, MD Chief Scientific Officer of Orchard Therapeutics talks about his company’s gene therapy in development to treat children with adenosine deaminase severe combined immunodeficiency (ADA-SCID). “These children are born without functional...
