Actively supporting advocacy efforts and engaging in novel clinical trials can provide patients with a sense of hope for potential therapies and assurance in the management of their condition. Read below about advancements made in June through clinical trials and events focused on inclusivity, equity, and advocacy for patients with rare liver disease.
GLI attends the PBC Foundation Summit, Edinburgh, Scotland
GLI participated in the PBC Foundation Summit, which served as an opportunity to delve into various aspects of PBC, including its impact on patient’s lives, advancements in research and treatment, and the significance of raising awareness and providing support. A few takeaways:
- Early diagnosis and intervention are imperative;
- Patients should be empowered through education and support systems;
- Research funding must be increased;
- Collaboration between healthcare professionals, researchers, and patient communities should be enhanced.
New Treatment Option for Cholestatic Pruritus in Alagille Syndrome Announced
With a greater demand for pruritus relief, patients diagnosed with Alagille syndrome and PFIC in the US welcome a new medication that recently received FDA approval for the treatment of cholestatic pruritus. Developed by Ipsen, a member of GLI’s Pediatric and Rare Liver Disease Council, Bylvay is now available by prescription. This news widens the array of treatment options, offering patients a chance to explore and determine which medication aligns best with their situation.
GLI Acknowledges the Achievements of Mirum and CANbridge in Pioneering a Global and Inclusive Clinical Trial Process for LIVMARLI
GLI commends the contributions made by Mirum and its collaborators in spearheading a comprehensive clinical trial process that prioritizes global accessibility and inclusivity for LIVMARLI, a drug developed for the treatment of cholestatic pruritus associated with Alagille Syndrome and PFIC. The substantial level of participation observed in this clinical trial for biliary atresia reflects a strong desire to find a cure.
GLI Commends Wilson’s Disease Association for Advocating for Affordability of Trientine Hydrochloride, a Generic Drug for Wilson’s Disease
Wilson’s Disease Association, a member of GLI’s Pediatric & Rare Diseases Council, in collaboration with the Mark Cuban Cost Plus Drug Company and Dr. Reddy’s Laboratories, has issued a formal statement emphasizing the significance of affordable pharmaceuticals for rare liver diseases. This statement specifically highlights the advancements and criticality of generic alternatives, such as trientine hydrochloride, as a substitute for Syprine. Cost-effective options will be made available for patients following this collaboration.
Upcoming Events
- July 13 – 15, 2023 – 43rd Aspen Conference on Pediatric Gastrointestinal Disease
- August 10, 2023 – Community Liver Alliance Rare Liver Disease Conference
- September 18 – 19, 2023 – Global Genes RARE Health Equity Forum
- September 19 – 21, 2023 – Global Genes RARE Advocacy Summit
A3: The Learning Experience will be held in Washington DC from September 30 – October 2, 2023!
If you are a liver patient, caregiver, family member, or clinician searching for an opportunity to become an effective advocate through interactive lessons on how to share your story, understand recent research & development, how to leverage social media and more.
For more information about the Pediatric and Rare Liver Diseases Council or to learn more about joining, please visit https://globalliver.org/pediatric-rare-liver-diseases-council/ or email pedsrare@globalliver.org.
