Concerted efforts are driving positive change in the pediatric and rare liver disease community! Read on for details:
Insight on Unmet Needs in Primary Biliary Cholangitis
In this Peer Exchange discussion on HCP Live, liver disease experts discussed the unmet needs related to primary biliary cholangitis (PBC). They identified that the primary issues were transplantation, ethnic disparities, and life-long PBC symptoms. Through this, a key insight emerged: While there are robust treatment options available, the challenge lies in raising disease awareness and simplifying information about diagnostic and treatment choices for patient understanding.
GLI Supports International PBC Day 2023
GLI is proud to support liver patients worldwide in recognition of International Primary Biliary Cholangitis (PBC) Day 2023. Annually observed on the second Sunday in September, International PBC Day serves as a platform to raise awareness about PBC. We especially highlight the work of the PBC Foundation as they will be holding a conference on PBC on September 9-10, 2023.
Find out more here: PBC Day 2023 Conference, Virtual Attendance Booking Form
Exciting Progress Made in Crigler-Najjar Therapy
Promising results were shown in a clinical trial using the gene therapy vector GNT0003 in a small sample of patients with Crigler-Najjar Syndrome. Like other rare liver diseases, patients suffering from Crigler-Najjar Syndrome often undergo liver transplantation to alleviate symptoms and manage the condition. With limited available organs and many barriers to transplantation, gene therapy and other alternatives hold great promise.
Increased Need for Insurance Coverage in Rare Liver Diseases
BC PharmaCare has extended its coverage to include medications for the rare diseases amyotrophic lateral sclerosis (ALS), acute hepatic porphyria (AHP), and long-chain fatty acid oxidation disorders (LC-FAODs). Similar strategies are in place across the country, assuring accessibility to high cost drugs. However, despite these measures, the cost of treatment remains a problem for those affected by their diseases.
A3: The Learning Experience
Washington DC, September 30 – October 2, 2023
Liver patients, their loved ones, and clinician advocates will be gathering in Washington, DC, at the end of the month to gain critical knowledge and skills for effective liver patient advocacy! Experts in the following topics will equip advocates to effect change in their communities: liver health basics, techniques in media and storytelling, the research and development process, health insurance and coverage, collaboration with policymakers, and more!
✍🏼Learn More
Upcoming Events
- September 18 – 19, 2023 – Global Genes RARE Health Equity Forum
- September 19 – 21, 2023 – Global Genes RARE Advocacy Summit
- September 31 – October 1, 2023 – Advanced Advocacy Academy
- October 2023 – #OctoberIs4Livers
- November 10 – 14, 2023 – AASLD Liver Meeting
For more information about the Pediatric and Rare Liver Diseases Council or to learn more about joining, please visit our webpage or email pedsrare@globalliver.org.
