Rare and Genetic Disease Network
Ron Cooper, President and CEO of Albireo, discusses drug development at Albireo, which has a clinical trial in progressive familial intrahepatic cholestasis (PFIC), a rare genetic condition that affects the liver.
Strongbridge Biopharma’s pivotal phase 3 SONICS study evaluating levoketoconazole (Recorlev) for the treatment of endogenous Cushing’s syndrome achieved statistical significance of its pre-specified primary endpoint.
Barry Greene, President of Alnylam, joined the company in 2003 and has more than 25 years of experience in healthcare, pharmaceutical, and biotechnology industries.
Gail Cawkwell, MD, PhD, Senior Vice President, Medical Affairs at Intercept, provides an overview of the company.
Yuval Cohen, PhD, CEO of Corbus Pharmaceuticals discusses his company’s growth over the past few years and provided an overview of Lenabasum, a novel, oral endocannabinoid-mimetic drug designed to resolve chronic inflammation and fibrotic processes.
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.
Richard West, Founder and CEO of Baebies, a newborn testing diagnostic company. In this video, Mr West discusses the company’s two main platforms.
Brian Schwartz, Chief Medical Officer of ArQule, discusses his company and it’s focus on disease management.
Heather A. Lau, MD, Director, Lysosomal Storage Disease Program at NYU Langone in New York City discusses the correlation between non-immune Hydrops fetalis (NIHF) and MPS VII.
Vanessa Vogel-Farley, Executive Director, Dup15q Alliance discusses Dup15q syndrome, the common name for chromosome 15q11.2-q13.1 duplication syndrome.
Barry Greene, President of Alnylam, provides RNA interference (RNAi) and the first ever new class of innovative medicines to potentially enter the market this year.
Acadia Pharmaceuticals and Neuren Pharmaceuticals announced that they have entered into an exclusive North American License Agreement for the development and commercialization of trofinetide for Rett syndrome and other indications.