by Peter Ciszewski | Nov 4, 2019
Patricia Weltin of Beyond the Diagnosis talks about Elhers-Danlos syndrome, a rare disease that afflicts two of her daughters. Ehlers-Danlos syndrome is a group of connective tissue disorders. There are various forms of Ehlers-Danlos. According to the NIH,...
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...
