Lessons learned in bringing novel options for rare liver diseases to patients and families

Patients with cholestatic liver diseases (CLD) do not have time to wait. Cholestatic liver diseases involve an impairment in bile formation or flow, which can profoundly impact patients’ health. One of the most debilitating symptoms associated with some CLDs is pruritus (insatiable itching) which can be so severe patients – especially infants and children – may scratch through their skin. Patients often turn to surgery, including liver transplant, to relieve the itching. However, like all major surgeries, liver transplants involve significant risks and nearly a quarter of liver transplants in children fail within the first six months, almost a third within five years, and almost half within 20 years.

Now, new treatments, including the first approved medicine for treating pruritus in progressive familial intrahepatic cholestasis (PFIC) patients, leveraging bile acid modulation with ileal bile acid transporter (IBAT) inhibitors are emerging that offer physicians novel, non-invasive options to add to their toolkit that help treat diseases and symptoms of diseases such as PFIC, Alagille syndrome (ALGS) and biliary atresia (BA), bringing new hope to patients and their families.

This webinar will highlight:
  • Rare cholestatic liver diseases, the science behind novel IBAT inhibition treatments and what the future holds for the treatment landscape
  • The impact of rare liver diseases on patients and caregivers
  • CLD leadership, global development and commercialization lessons learned from Albireo, a global rare liver disease company focused on the development of novel bile acid modulators to treat pediatric cholestatic and adult liver diseases, who recently brought a new treatment to market
  1. Srivastava A. J Clin Exp Hepatol 2014;4:25-36.
  2. Bjørnland K et al. Eur J Pediatr Surg. 2020.
  3. van Wessel DBE et al. Hepatology. 2021;10.1002/hep.31787.
  4. Venick et al. J Am Coll Surg. 2018;226(4):355-366
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Ron Cooper

Ron Cooper

President and CEO, Albireo Pharma

Ron Cooper, Albireo’s first President and CEO, is a global P&L leader with a track record of growing businesses, brands, and organizations in the U.S. and Europe. Since joining Albireo, Ron has been instrumental in growing the business to a global biopharmaceutical company and executing the global launch of Bylvay® (odevixibat), the first drug treatment for patients with progressive familial intrahepatic cholestasis (PFIC).

For over 25 years, Ron worked in five different countries and held positions of increasing responsibility in sales, marketing, and general management with Bristol-Myers Squibb, culminating in his role as President of Europe where he was responsible for over 30 countries with sales exceeding $4.5 billion. Ron has successfully completed more than a dozen business development deals, including the creation of the first single tablet HIV/AIDS regimen partnership. He is a graduate of St. Francis Xavier University in Canada.

Nadia Ovchinsky

Nadia Ovchinsky

Pediatric Gastroenterologist and Hepatologist, Children’s Hospital at Montefiore and ASSERT Principal Investigator

Nadia Ovchinsky, MD, MBA, FAALSD is the Director of Pediatric Hepatology, Medical Director of Pediatric Liver Transplantation, and a Professor of Pediatrics at Albert Einstein College of Medicine - Children's Hospital at Montefiore. Dr. Ovchinsky received her MD and MBA in Healthcare Management from Rutgers-Robert Wood Johnson Medical School. She completed her pediatric residency, pediatric gastroenterology fellowship, as well as advanced training in pediatric transplant hepatology at Columbia University - Morgan Stanley Children's Hospital of New York-Presbyterian. Dr. Ovchinsky’s clinical and research interests involve improving quality and enhancing the delivery care for children with chronic liver disease, optimizing outcomes of pediatric liver transplantation, and advancing therapies for children with cholestatic liver disorders. Dr. Ovchinsky is an internationally known expert in pediatric liver disease and is an author of numerous journal articles, invited reviews, webinars, and book chapters. Dr. Ovchinsky is actively involved in public policy and advocacy efforts to benefit children with liver disease. She has served in leadership positions with professional societies such as the North American Society of Pediatric Gastroenterology, Hepatology, and Nutrition, American Liver Foundation, and American Association for the study of Liver Disease. Dr. Ovchinsky has been recognized with numerous academic and humanism in medicine awards, as well as teaching awards for her commitment and dedication to the education of medical students, residents, and fellows.

Cher Bork

Cher Bork

Executive Director, Alagille Syndrome Alliance

Born and raised in Memphis, TN, Cher started fundraising for St. Jude Children’s Research Hospital when she was around 10 years old, holding car washes and block parties and volunteering to serve meals at the local mission. That early exposure to the need around her established a deep passion for service, and those with critical illness truly touched her heart. After her own personal journey with a rare disease, she was set on the path to make a difference with a cure-based organization. Joining the ALGSA in January 2018 as the Development Manager, she has had the privilege of developing relationships with ALGS families, industry, and the rare disease community. In October 2018, the Board of Directors appointed her as Executive Director. With a long history in nonprofit fundraising, event management and development, as well as serving on nonprofit boards including Boys & Girls Club, she is committed to leading ALGSA toward future growth, sustainability, and their goal: to find better treatments, a cure, and a better life for those impacted by Alagille Syndrome.


Arsalan Arif

Founder & Publisher, Endpoints News

Arsalan Arif is a news media entrepreneur who set out in 2015 to build his vision of an independent biotech news company at Endpoints News.