During May, significant strides were made towards prioritizing the diagnosis and treatment of rare liver diseases. This month, we are excited to share some innovative approaches to enhance the diagnostic and treatment processes of rare liver diseases, which have traditionally been overlooked and lacked appropriate medical interventions.
GLI Applauds Congress for Urging FDA to Review Rare Disease Therapy Procedures, A Major Step Towards Better Treatment Options
With numerous liver diseases lacking effective treatment options, GLI is encouraged to see the Congressional Rare Disease Caucus advocating for the FDA to prioritize rare diseases on their agenda. Specific initiatives that were mentioned include assessing inconsistencies in rare disease policies, reviewing the implementation of approval pathways, and providing increased clarity for employees to ensure continuity of in-process application reviews. We commend the Caucus’s work in promoting a more consistent and reliable approval process for these essential medications.
Significant Progress Made in Streamlining Diagnosis of Wilson’s Disease in China
Wilson’s disease is a rare genetic disorder that causes copper to build up in the body, leading to various neurological and liver-related symptoms. Due to its variable symptoms that can overlap with other conditions and the need for specialized tests to measure copper levels, diagnosis can be challenging. However, a recent study published in Liver Research suggests that a novel and comprehensive nomogram based on routine clinical indicators, which can be obtained through noninvasive methods, may improve the diagnosis of Wilson’s disease in the future. This streamlined diagnostic process, using six routine blood indicators, is expected to reduce the likelihood of missing or misdiagnosing the disease.
Upcoming Report of Ocaliva and bezafibrate as promising treatment option for Primary Biliary Cholangitis (PBC)
GLI is looking forward to the presentation of Phase II trial data and research by Ipsen, one of our council members, at EASL in June 2023. They will be sharing their findings on the utilization of Ocaliva in conjunction with bezafibrate for the treatment of PBC. This significant development, coupled with the FDA’s grant of orphan drug status, holds great promise for the future of PBC treatment.
GLI attends the BARE Inc. Symposium, Chicago, IL
GLI joined patients, advocates, and healthcare providers in Chicago for BARE Inc’s first Biliary Atresia Symposium on May 5-6, 2023. Key focal points included emphasizing early diagnosis in pediatric liver diseases, highlighting breakthroughs in research and treatment, recognizing the importance of collaboration and multidisciplinary care, advocating for patient-centered approaches, and addressing challenges related to access to care and health equity. The overarching objective was to enhance the well-being of affected children and advance the global comprehension and management of rare liver diseases.
Upcoming Events
- June 8, 2023 – International NASH Day
- June 21-24, 2023 – EASL Congress 2023; GLI will be in Vienna, pass by our stand to say hello or email info@globalliver.org to set up a meeting with us.
- July 13-15, 2023 – 43rd Aspen Conference on Pediatric Gastrointestinal Disease
- August 10, 2023 – Community Liver Alliance Rare Liver Disease Conference
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.
