The Food and Drug Administration (FDA) approved Alnylam Pharmaceuticals’ patisiran (Onpattro) infusion for the treatment of polyneurotherapy caused by hereditary transthyretin-mediated amyloidosis (hATTR) in adult patients.
Gail Cawkwell, MD, PhD, Senior Vice President, Medical Affairs at Intercept, discusses the challenges of diagnosing and treating primary biliary cholangitis (PBC), a rare liver disease that is caused by an autoimmune reaction.
Richard West, Founder and CEO of Baebies, discusses the public health program of newborn screening.
The Food and Drug Administration (FDA) approved Azedra (iobenguane I 131) injection for intravenous use for the treatment of adults and adolescents age 12 and older with rare tumors of the adrenal gland (pheochromocytoma or paraganglioma) that cannot be surgically removed (unresectable), have spread beyond the original tumor site and require systemic anticancer therapy.
Mr. Robert (Bob) E. Ward, Chairman of the Board and Chief Executive Officer of Eloxx Pharmaceuticals discusses Right To Try, which is aimed at allowing terminally ill Americans to try medicines that have passed Phase 1 of the FDA approval process and remain in clinical trials but are not yet on pharmacy shelves.
Heather A. Lau, MD, Director, Lysosomal Storage Disease Program at NYU Langone in New York City discusses Fabry disease, an inherited disorder that results from the buildup of a particular type of fat, called globotriaosylceramide, in the body’s cells.