by Peter Ciszewski | Dec 1, 2021
Anne Pariser, MD, Director of the NCATS’ Office of Rare Diseases Research (ORDR), provides an overview of the ORDR and the research they are involved with. The ORDR is focused on multiple programs to improve the efficacy of rare disease research. Their two...
by Peter Ciszewski | Nov 30, 2021
Pamela Vig, PhD, Chief Scientific Officer, Mirum Pharmaceuticals, gives a detailed overview of Alagille syndrome and the recent presentations at The Liver Meeting 2021 highlighting the efficacy maralixibat to treat this rare disorder. As Dr. Vig explains,...
by Peter Ciszewski | Nov 30, 2021
Evan S. Dellon, MD, MPH, Gastroenterologist and Professor of Medicine and Adjunct Professor of Epidemiology at the University of North Carolina Chapel Hill, discusses treatment strategies for eosinophilic esophagitis (EoE) and describes the mechanism of action...
by Peter Ciszewski | Nov 29, 2021
Fennec Pharmaceuticals announced today that it expects to receive a Complete Response Letter (CRL) from the U.S. Food and Drug Administration for Pedmark (sodium thiosulfate), an investigational treatment for the prevention of ototoxicity associated with cisplatin...
by Peter Ciszewski | Nov 29, 2021
Anthony Quinn, PhD, President and CEO of Aeglea BioTherapeutics, discusses the PEACE study, a phase 3 clinical trial of pegzilarginase for the treatment of arginase 1 (ARG1) deficiency. ARG1 deficiency is a rare genetic disorder characterized by complete or...
