Research in Primary Biliary Cholangitis

  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...

The Success of the Orphan Drug Act

  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...

Considering Rare Diseases for Challenging Cases

  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)...

Challenges of the Health Care System Supporting Innovative Drugs

  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...
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