Rare and Genetic Disease Network
Featured
Rare Disease Drug Development at Albireo
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.
Positive Results in Phase 3 Cushing’s Syndrome Trial
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.
Alnylam Overview
Barry Greene, President of Alnylam, joined the company in 2003 and has more than 25 years of experience in healthcare, pharmaceutical, and biotechnology industries.
Interecept Pharma: Focused on Non-Viral Liver Diseases
Gail Cawkwell, MD, PhD, Senior Vice President, Medical Affairs at Intercept, provides an overview of the company.
Corbus Pharmaceuticals Corporate Overview
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.
Patisiran: First RNAi Drug Approved to Treat Polyneuropathy Caused By hATTR
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.
Baebies: Newborn Screening Platforms
Richard West, Founder and CEO of Baebies, a newborn testing diagnostic company. In this video, Mr West discusses the company’s two main platforms.
ArQule’s Focus on Rare Diseases
Brian Schwartz, Chief Medical Officer of ArQule, discusses his company and it’s focus on disease management.
Correlation Between NIHF and MPS VII
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.
Dup15q Syndrome Overview
Vanessa Vogel-Farley, Executive Director, Dup15q Alliance discusses Dup15q syndrome, the common name for chromosome 15q11.2-q13.1 duplication syndrome.
Alnylam’s New RNAi Therapy
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.
New License Agreement for the Development of Trofinetide in Rett Syndrome
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.
Special Reports
Rare in Common Podcast
Considering Rare Diseases for Challenging Cases
Duchenne Muscular Dystrophy
Rare Disease Diagnosis: Huntington’s Disease
Rare in Common Podcast
Considering Rare Diseases for Challenging Cases
Duchenne Muscular Dystrophy
