In the rare liver disease space, education for healthcare providers and researchers is just as important as awareness, patient and caregiver education, and patient advocacy.
First administration of gene therapy clinical trial for Wilson’s Disease completed at Yale New Haven Health
Wilson’s disease is a rare genetic disorder that causes copper to accumulate in the body, leading to liver and neurological damage. A gene therapy clinical trial that uses a virus to deliver a corrected version of the gene that is defective in Wilson’s disease was administered to the very first patient. Early results suggest that the therapy is safe and well-tolerated. If successful, this approach could offer a promising new symptom management option for patients with Wilson’s disease.
Gaining momentum for liver care: Arun Sanyal reflects on the VCU liver institute’s first year
The newly established Liver Disease and Metabolic Health Institute at Virginia Commonwealth University (VCU) presents significant opportunities for teaching and learning in liver disease. Since December 2021, the Institute has focused on transformative innovations in the prevention, diagnosis, and treatment of liver disease, with the ultimate goal of improving patient outcomes. In its first year, the Institute has already begun to make progress and its leadership looks forward to expanding its educational offerings to encompass the rare liver disease landscape in the future. We are excited to see what’s next for the VCU liver institute and hope that other institutions will follow suit in prioritizing research in liver disease.
Total robotic liver transplant surgery saves 8-month-old toddler
In Mumbai, a complete robotic liver transplant was performed at the Global Hospital on an 8-month old with Crigler Najjar Syndrome. The complete robotic surgical system provided high precision and control – which allowed for a reduction in blood loss, lower risk of infection, and less postoperative complications. The child was discharged only 3 days after the relatively quick transplant. Robotic surgery can be effective at all ages for liver transplant and may be considered for other organs as well.
Genethon given PRIME status by EMA for gene therapy to treat Crigler-Najjar Syndrome
The Europeon Mediciens Agency has granted the drug GNT-0003 as a PRIME or Priority medicines status as of March 7, 2023. This is currently the only clinical trial and drug that has been shown to have significant therapy on Crigler-Najjar syndrome, a disease that affects the levels of bilirubin in the blood. Severe cases can lead to neurological damage and death. As a rare liver disease, it is essential for physicians to learn about the disease and access ways for patients to manage their disease.
Upcoming Council Member Events
- May 1-3, 2023 – Global Genes RARE Drug Development Symposium
- May 5-6, 2023 – BARE Inc. Symposium
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.