by Peter Ciszewski | Oct 25, 2018
At this year’s American Academy of Child and Adolescent Psychiatry (AACAP) Annual Meeting in Seattle, Washington, October 22-27, Ovid Therapeutics announced additional efficacy data from the company’s Phase 2 STARS trial that further support the potential of...
by Peter Ciszewski | Oct 25, 2018
Pharnext announced positive topline results from its pivotal Phase 3 clinical trial (PLEO-CMT) evaluating two doses of PXT3003 compared to placebo during 15 months for the treatment of Charcot-Marie-Tooth type 1A disease (CMT1A). PLEO-CMT was a pivotal, 15-month,...
by Peter Ciszewski | Oct 25, 2018
The U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to lead product candidate A4250, an ileal bile acid transporter (IBAT) inhibitor, for the treatment of Alagille syndrome, a rare and life-threatening disease that affects the liver and has no...
by Peter Ciszewski | Oct 23, 2018
Barry Ticho, MD, Chief Medical Officer at Stoke Therapeutics, provides an overview of Dravet syndrome, a severe form of epilepsy that is part of a group of diseases known as SCN1A-related seizure disorders. Dravet syndrome usually appears during the first year...
by Peter Ciszewski | Oct 22, 2018
Richard A. Basile, Co Founder and CEO of BioPontis Alliance for Rare Diseases, discusses the state of rare diseases and the critical challenges facing it. BioPontis is an international nonprofit organization whose mission is to advance promising science into...
