Platinum Sponsors
Ipsen is a global biopharmaceutical company focused on innovation and specialty care. At Ipsen, we develop and commercialize medicines in three key therapeutic areas – Oncology, Neuroscience and Rare Disease. We work to provide hope for people whose lives are impacted by rare liver diseases, such as Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC), a rare growth disorder or rare bone disease. To us, it’s important we put the person living with rare disease first in all that we do. Every day, our more than 5,000 employees worldwide, including nearly 800 in North America, work diligently to improve lives around the world.
Gold Sponsor
Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare diseases affecting children and adults.
LIVMARLI, an IBA¬¬T inhibitor, is approved for the treatment of cholestatic pruritus in patients with ALGS in the U.S. (3 months+), in Europe (2 months+), and in Canada. Cholbam is FDA-approved for the treatment of bile acid synthesis disorders due to single enzyme deficiencies and adjunctive treatment of peroxisomal disorders in patients who show signs or symptoms or liver disease. An sNDA for LIVMARLI has also been submitted in the US for pruritus due to PFIC.
Mirum’s late-stage pipeline includes a Phase 2b study evaluating LIVMARLI in biliary atresia. A second IBAT inhibitor, volixibat, has two ongoing Phase 2b studies, VISTAS for PSC and VANTAGE for PBC. Chenodal is being evaluated in an ongoing Phase 3 study, RESTORE, for cerebrotendinous xanthomatosis.
Learn more at mirumpharma.com.
Silver Sponsor
Alcresta develops novel products that deliver enzymatic solutions to those living with rare diseases. Based in Massachusetts, Alcresta Therapeutics has achieved significant commercial milestones, created an extensive intellectual property portfolio, optimized production capabilities, and developed exciting scientific data supporting its products and technology platform.
Children’s Health℠ is the leading pediatric health care system in North Texas and has long been recognized as a leader in pediatric health. Children’s Health campuses include Children’s Medical Center Dallas, Children’s Medical Center Plano and multiple Children’s Health Specialty Centers. With its academic partner, UT Southwestern, Children’s Medical Center Dallas is consistently ranked the #1 children’s hospital in North Texas and among the nation’s best pediatric hospitals by U.S. News & World Report. Its commitment to excellence and providing outstanding care across all aspects of pediatrics has resulted in being ranked across all specialty programs for seven consecutive years, including Cancer, Cardiology & Heart Surgery, Behavioral Health, Diabetes & Endocrinology, Gastroenterology & GI Surgery, Neonatology, Nephrology, Neurology & Neurosurgery, Orthopedics, Pulmonology, and Urology.
A pioneer in its field, Echosens significantly changed the practice of liver diagnosis with FibroScan®, the non-invasive gold standard solution for comprehensive management of liver health. FibroScan® is recognized worldwide as the reference for liver fibrosis and liver steatosis assessment with more than 5,384+ peer-reviewed publications and 218+ international guidelines.
Fresenius Kabi is a global company specializing in lifesaving medicines and technologies for infusion, transfusion, and clinical nutrition.
Gilead Sciences is a biopharmaceutical company focused on liver disease, HIV, and viral treatments
Pierre Fabre Pharmaceuticals is on a mission to find new ways to care and deliver breakthrough therapies in oncology and rare diseases to patient populations with high unmet needs and limited treatment options.
A global biopharmaceutical leader advancing treatments in gastroenterology, rare diseases, and immunology.
Bronze Sponsor
Specializes in organ preservation solutions and technologies supporting transplant outcomes.
Developing cellular biomarkers for personalized diagnosis and drug development in infectious diseases, solid organ transplantation, as well as immunological disorders.
Hospitals / Centres
Leading pediatric hospital offering liver and multi-organ transplant care in Central Florida.
Lurie has performed 500+ pediatric liver transplants and leads the Great Lakes region. Their program includes advanced techniques like split/reduced-size and living donor transplants.
Here, it’s different.
The Liver Transplant Program at Children’s Hospital Colorado offers a unique and specialized program devoted to the care of children who require liver transplantation. Our unique multidisciplinary team cares for kids and families from the time of diagnosis to transplant and all the way through long-term post-transplant care. We achieve some of the best outcomes in the nation by working with every medical and surgical specialty in the hospital to reimagine children’s lives.
World-renowned children’s hospital with extensive transplant research and services.
The Brendan Tripp Elam Transplant Center at Children’s Mercy Kansas City offers pediatric heart, liver, kidney, and multi-organ transplants. It includes a Level I pediatric trauma center and a nationally recognized NICU, with over 8,400 employees and more than 50 specialties.
The Pediatric Liver Transplant team at Children’s of Alabama has collaborated with the University of Alabama at Birmingham (UAB) transplant services since 1993 and has performed 150 liver transplants. The Pediatric Transplant Center at Children’s of Alabama was opened in March 2013 at the Benjamin Russell Hospital for Children. Our patients are cared for by a skilled, multidisciplinary team including UAB surgeons, pediatric gastroenterologists, hepatology services, liver transplant coordinators, and other specialists in our transplant program. Our team coordinates all aspects of care during the evaluation phase, transplantation, and post-procedure care to ensure positive outcomes.
Since 1911, Children’s of Alabama has provided specialized medical care for ill and injured children. Children’s of Alabama is the only medical center in Alabama dedicated solely to the care and treatment of children. It is a private, not-for-profit medical center that serves as the teaching hospital for the UAB pediatric medicine, surgery, psychiatry, research and residency programs.
Children’s Wisconsin has been a pioneer in pediatric heart, liver, kidney and bone marrow transplants for over three decades, helping hundreds of children with end-stage diseases survive and thrive. They offer many family support services including an Access Center, Child Life Services, and a Family Resource Center.
Cincinnati Children's is a nonprofit pediatric health system, consistently ranked among America's best hospitals and a top recipient of NIH pediatric research funding.
MedStar Health—It’s how we treat people
Transplant medicine that changes lives
MedStar Georgetown Transplant Institute offers the complete spectrum of care from evaluation and medical management to cutting-edge surgery and transplant procedures. We provide hope and life-restoring treatments for patients with end-stage organ failure.
The Institute is one of the highest volume organ transplant programs in the country and offers the expertise of some of the foremost US transplant specialists. We have performed more than 8,500 liver, kidney, pancreas, small bowel, and multi-organ transplants. And our volumes speak volumes. For all organs, our one-year adult and pediatric survival rates are among the best in the country.
University of Michigan’s children’s hospital with strong transplant expertise.
Nemours is one of the largest multistate pediatric systems, with two children's hospitals and 70+ care practices, focusing on holistic, high-quality child health.
Phoenix Children’s is Arizona’s only freestanding comprehensive pediatric hospital, and has grown to become one of the largest children’s hospitals in the United States. With a medical staff of nearly 1,000 pediatric specialists, Phoenix Children’s provides inpatient, outpatient, trauma and emergency care across more than 70 pediatric subspecialties, the most comprehensive pediatric care available in the state.
The pediatric liver transplant program at Phoenix Children’s provides an invaluable resource to the state of Arizona and the local community as the only pediatric liver transplant program in Arizona. The program uniquely integrates a multidisciplinary liver transplant team approach that is equipped to meet the needs of every child at each stage of development. In addition to pediatric transplant physicians and transplant surgeons, the team includes specialists in child life, social work, nutrition, pharmacology, and transplant nursing coordination to ensure continuous care and management of patients through every period of a successful liver transplant.
Utah’s major pediatric transplant center, affiliated with the University of Utah.
With 50+ years of transplant experience, Stanford leads in combined heart-liver and other multi-organ pediatric transplants, and is #1 nationally in pediatric kidney transplant volume.
Since 1985, St. Louis Children’s has performed over 400 liver transplants including dual-organ and split-liver procedures, with a strong living donor program.
Texas Children’s leads the U.S. in liver transplants by volume and complexity, having performed nearly 800 since 1988 and accepting more organ offers than any other program.
The SickKids Transplant and Regenerative Medicine Centre (TRMC) is Canada's most research-intensive paediatric transplant program and the largest centre dedicated to comprehensive transplant care in Canada.
Our case volumes are now within the top five per cent of centres in North America, and our Hospital’s survival rates match or exceed the best programs. With a staff that includes professionals from all disciplines of health care and research, the TRMC provides the best in complex and specialized care by creating scientific and clinical advancements, sharing knowledge and expertise, and advocating on behalf of children who need transplant care.
As innovators in transplantation, the TRMC improves the health of children by seamlessly integrating care, research, and teaching.
Life Changing Medicine
As a nationally recognized center of excellence for pediatric transplantation, UPMC is a leader in pediatric liver transplant specializing in living donor transplant as well as transplants for those with metabolic diseases. Our pediatric hepatology and transplant services are always available for consultation. Pediatric transplant hepatology and transplant surgical fellowship training is offered by our teams. UPMC also directs a pediatric liver transplant network in collaboration with UVA Charlottesville, Advent Health Orlando and Atrium Health Charlotte, N.C.
Non-Profit
The National Living Donor Assistance Center's mission is to reduce the financial disincentives to living organ donation. To this end, we operate a nationwide system that provides reimbursement of travel and subsistence expenses, lost wages, and dependent care expenses to people being evaluated for and/or undergoing living organ donation.
ALF’s mission is to promote education, advocacy, support services and research for the prevention, treatment and cure of liver disease.
Patient Advocacy
At ALGSA, we lead with purpose and heart—delivering vital support, trusted resources, and a strong sense of community to those facing this complex, rare genetic disorder. Our commitment is grounded in improving the lives of ALGS patients and their loved ones through collaboration, education, and empowerment. We are deeply engaged in science and research, working tirelessly to drive initiatives that uncover real answers, improve treatments, and ultimately lead to a cure. Through our research network and programs, we bring together all key stakeholders—physicians, scientists and researchers, biotech and pharmaceutical companies, patients and families, and other nonprofits facing similar medical challenges—to advance knowledge, fill critical gaps, and keep the patient voice front and center.
BARE was created to help solve some of the deficiencies and frustrations that currently exist in the Biliary Atresia community. We believe creating an organization dedicated solely to Biliary Atresia, will help push for progress in finding a cause, treatment, and one day, a cure.
A global platform convening leading experts and patients to discuss innovations and unmet needs in the field of cholestatic liver disease.
Primary Sclerosing Cholangitis (PSC) is a rare liver disease that damages the bile ducts inside and outside the liver. With PSC, bile ducts become inflamed, and the inflammation leads to scarring and narrowing of the affected ducts. Eventually, blockages may occur. As the scarring blocks more and more ducts, bile becomes trapped in the liver. This damages the liver and can result in fibrosis, cirrhosis, and liver failure. Patients may eventually require a liver transplant.
Progressive Familial Intrahepatic Cholestasis (PFIC) is an umbrella term that represents a group of rare genetic disorders that cause cholestatic liver disease and can lead to cirrhosis and end-stage liver disease.
We offer a variety of educational resources designed to help families understand this complex disease including a patient brochure translated into 19 languages and a library of webinars from PFIC experts.