Gail Cawkwell, MD, PhD, Senior Vice President, Medical Affairs at Intercept, discusses current research in primary biliary cholangitis (PBC), a rare liver disease that is caused by an autoimmune reaction. When bile is not able to move through the bile ducts, it...
Mr. Robert (Bob) E. Ward, Chairman of the Board and Chief Executive Officer of Eloxx Pharmaceuticals discusses the success of the Orphan Drug Act (ODA). This Act was first enacted in the United States in 1983 and set up to encourage the development of drugs for...
There seems to be some good news on the horizon for individuals with Charcot-Marie-Tooth (CMT) disease. Acceleron Pharma announced positive preliminary results from Part 1 of the Phase 2 clinical trial with ACE-083 in patients with CMT disease at the Peripheral Nerve...
Heather A. Lau, MD, Director, Lysosomal Storage Disease Program at NYU Langone in New York City discusses how physicians need to consider rare diseases for some of their more challenging diagnositic cases. Rare diseases are problematic on many fronts: (1)...
The U.S. Food and Drug Administration (FDA) approved Tibsovo (ivosidenib) tablets for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) who have a specific genetic mutation. This is the first drug in its class (IDH1 inhibitors)...
Editor’s Note: This interview was conducted at #Bio2018. Please pardon the background noise. Chris Garabedian discusses the challenges of our health care system in supporting the many emerging and innovative therapies entering the market. Chris also...
