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– Calls to unite global communities affected by rare diseases –
– Albireo supports increasing education and advances in research for rare cholestatic liver diseases –
BOSTON, Feb. 26, 2021 (GLOBE NEWSWIRE) -- Albireo Pharma, Inc. (Nasdaq: ALBO), a clinical-stage rare liver disease company developing novel bile acid modulators, joins the rare disease community in support of Rare Disease Day 2021. Established by EURORDIS, Rare Disease Day www.rarediseaseday.org aims to build broader awareness of the unique needs and challenges of patients and families affected by rare diseases. This year’s theme of Stay Connected focuses on helping rare disease communities around the world share information and support online with the first fully digital and interactive events planned by organizations in patient advocacy, research and patient care around the world.
“Within the general population, very little is known about rare childhood liver conditions. On Rare Disease Day, the voices, experiences, struggles and milestones of these communities take center stage,” said Alison Taylor, Chief Executive of Children’s Liver Disease Foundation (CLDF). “This is a day to share stories and educate professionals, families and young people, and this platform brings rare disease groups together in an otherwise very isolating environment.”
Through greater connectivity with the patient community, Albireo will further the mission to advance life-changing research that can lead to new treatment options and better care, all informed by the patient voice. Albireo will be spotlighting rare patient communities and the critical needs of patients, caregivers and the medical community around the world. To Stay Connected, the Company is launching a new @PFICvoices Instagram page to connect patients and families who are managing progressive familial intrahepatic cholestasis (PFIC), a rare, life-threatening disease. Members of the global PFIC community can share personal stories and experiences. Patient and caregiver stories will also be featured on the PFIC Voices Facebook page and Albireo Twitter and LinkedIn channels, and supportive educational resources in seven languages can be found at pficvoices.com/resources/.
“The experiences of advocates, patients and families inspire and guide us in our work to advance research targeting new treatments for cholestatic liver diseases, including PFIC, Alagille syndrome, biliary atresia and PSC,” said Ron Cooper, President and Chief Executive Officer of Albireo. “Staying connected with the patient community and their stories impacts everything we do, from our patient education resources to our clinical trials, with the ultimate goal of potentially reducing the burden of rare cholestatic liver diseases.”
For information on patient advocacy organization and disease education, check out these great resources: CLDF at childliverdisease.org; the PFIC Advocacy and Resource Network at PFIC.org; Alagille Syndrome Alliance at Alagille.org; and PSC Partners and Seeking a Cure at pscpartners.org.
Progressive familial intrahepatic cholestasis (PFIC) is a rare disorder that causes progressive, life-threatening liver disease. Patients have impaired bile flow, or cholestasis, caused by genetic mutations. The resulting bile build-up in liver cells causes liver disease and symptoms. The most prominent and problematic ongoing manifestation of the disease is pruritus, or intense itching, which often results in a severely diminished quality of life. Other symptoms include jaundice, poor weight gain and slowed growth. In many cases, PFIC leads to cirrhosis and liver failure within the first 10 years of life, and nearly all people with PFIC require treatment before age 30. There are no drugs currently approved for PFIC, only surgical options that include partial external biliary diversion (PEBD) and liver transplantation. Additional information on PFIC is available at https://www.pficvoices.com.
Alagille syndrome (ALGS) is a rare, multisystem genetic disorder that can affect the liver, heart, skeleton, eyes, central nervous system, kidneys and facial features. Liver damage is caused by a paucity of bile ducts preventing bile flow from the liver to the small intestine. Approximately 95% of patients with ALGS present with chronic cholestasis, usually within the first three months of life, and up to 88% also present with severe, intractable pruritus. Currently, there are no approved drug treatments.
About Biliary Atresia
Biliary atresia is a rare pediatric liver disease with symptoms typically developing about two to eight weeks after birth. Damaged or absent bile ducts outside the liver result in bile and bile acids being trapped inside the liver, quickly resulting in cirrhosis and even liver failure. Children have clay-colored or no color in their stools and jaundice, among other things, and a few patients are pruritic. Biliary atresia is the most common pediatric cholestatic liver disease and is the leading cause of liver transplants among children as there are no approved drug treatments.
Albireo Pharma is a clinical-stage biopharmaceutical company focused on the development of novel bile acid modulators to treat rare pediatric and adult liver diseases. Albireo’s lead product candidate, odevixibat, is being developed to treat rare pediatric cholestatic liver diseases with Phase 3 pivotal trials in PFIC, Alagille syndrome and biliary atresia. The Company completed IND-enabling studies for new preclinical candidate A3907 and plans to advance development in adult liver disease. Albireo was spun out from AstraZeneca in 2008 and is headquartered in Boston, Massachusetts, with its key operating subsidiary in Gothenburg, Sweden. The Boston Business Journal named Albireo one of the 2020 Best Places to Work in Massachusetts for the second consecutive year. For more information on Albireo, please visit www.albireopharma.com.