International Alagille Awareness Day
The very first International Alagille Awareness day was held on January 24th, 2020.
What is Alagille syndrome? It is a rare disease which effects the heart, liver, eyes, and skeletal system. Unfortunately, there is yet to be an approved therapy for Alagille. The syndrome was named for the man who was the first to describe it, Dr. Daniel Alagille, who was a researcher and a pediatric hematologist.
The new awareness day will help to not only make the public more aware of the syndrome, but it will also serve as a day of support for all those affected by it.
The Alagille Syndrome Alliance (ALGSA) is an international nonprofit organization dedicated to supporting Alagille syndrome patients and their families. They, along with Albireo Pharmaceuticals, are together celebrating this new awareness day.
Cher Bork is the Executive Director of ALGSA. The organization sponsors research grants for Alagille, promotes awareness, facilitates community education programs (through in person as well as digital means), and directly assists families. This organization understands that it is not just about spreading knowledge to patients; it must be shared among healthcare professionals, researchers, and the general public.
Albireo, founded in 2008, focuses on rare pediatric cholestatic liver diseases. Their lead product is called odevixibat. This therapy is specifically in development for progressive familial intrahepatic cholestasis. It is currently in Phase 3 of development for this indication.
They have already elobixibat approved for chronic constipation in Japan. This is the very first IBAT inhibitor that has ever been approved in ANY country.
Two other Phase 2 products are in the works. Additionally, they have plans to continue development for odevixibat as a therapy for other conditions, including potentially Alagille syndrome.
Their goal, like everyone in the rare community, is to find cures and therapies for rare patients which will significantly enhance their quality of life.
You can read more about this new awareness day here.