It’s Rare Liver Diseases Month!
If you haven’t already, join us for the 2024 #RareAware multifaceted campaign, focusing on the theme of “Transitions in Care”:
- Hear from patients and caregivers during weekly #GLILIVE conversations on Wednesdays at 12pm ET.
- Join our Empowerment Day virtual seminar for pediatric patients (ages 8-18), on February 17, 2024, which will connect individuals who have successfully navigated care transitions with those currently undergoing similar journeys. Register here.
- Help us spread awareness about rare liver diseases by sharing graphics on social media using our toolkit.
- Show your support by checking out our merch!
Learn more about the campaign by reading our press release or checking our website.
Congratulations to the Children’s Hospital Los Angeles on their 500th Liver Transplant
In January of this year, the Children’s Hospital Los Angeles (CHLA) marked a significant milestone by completing its 500th liver transplant. Since their first liver transplant in 1998, CHLA has progressed to achieve a one-year patient and graft survival rate of 100%.
Celebrating VUMC’s First Combined Lung and Liver Transplant
Vanderbilt University Medical Center (VUMC) reached a significant milestone by conducting its first combined lung and liver transplant on a patient with a rare autoimmune disease known as sarcoidosis. This accomplishment highlights that multidisciplinary care is crucial in the complex, innovative solutions to complex conditions.
mRNA Therapies as a Potential Therapy for Argininosuccinic Aciduria
A recent study using a mouse model has shown the potential effectiveness of messenger RNA (mRNA) therapies for treating the rare liver genetic disease argininosuccinic aciduria (ASA). ASA is an inherited metabolic disorder that affects how the body breaks down protein. ASA can lead to liver dysfunction and damage due to high levels of ammonia accumulating in the liver and bloodstream. Further exploration of this mRNA therapy offers hope for addressing not only ASA but also other rare, incurable genetic diseases.
Exciting Collaboration on the Horizon for Rare Liver Disease Gene Therapy
Swiss biotech company Genevant Sciences and gene editing firm Tome Biosciences have joined forces to address an undisclosed monogenic liver disease. Their collaboration seeks to combine Genevant’s lipid nanoparticle technology with Tome’s programmable genomic integration technology, aiming to create an in vivo gene editing treatment. This partnership is an exciting application of novel techniques for rare liver disease patients!
Upcoming Events
- February – Rare Liver Diseases Month, #RareAware Campaign
- February 17, 2024 – #RareAware Empowerment Day, virtual seminar for pediatric patients
- February 25 – 28, 2024 – EveryLife Foundation Rare Disease Week on Capitol Hill
- February 28, 2024 – Community Liver Alliance Rare Liver Disease Virtual Program for Patients and Caregivers
- February 29, 2024 – Rare Disease Day at NIH
- March 1, 2024 – FDA Rare Disease Day Public Meeting
- April 23 – 25, 2024 – World Orphan Drug Congress USA 2024
- April 29 – May 1, 2024 – Global Genes RARE Drug Development Symposium
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.
