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.

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.

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.

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.

Special Reports