Albireo Spotlights Global Patient Communities on Rare Disease Day
February 26 2021 - 8:30AM
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.
About PFICProgressive 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.
About ALGSAlagille 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 AtresiaBiliary 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.
About AlbireoAlbireo 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.
Media Contacts:Colleen Alabiso,
857-356-3905, colleen.alabiso@albireopharma.comLisa Rivero,
617-947-0899, lisa.rivero@syneoshealth.com
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