Rare Hepa Pedi

06/12/2025

02/05/2024

A novel treatment strategy utilizing panobinostat for high-risk and treatment-refractory hepatoblastoma

Hepatoblastoma (HB) is the most prevalent liver cancer in children, yet treatment options are limited, resulting in survival rates of less than 50%. This study in the Journal of Hepatology demonstrates that a triple combination of the pan-HDAC inhibitor Panobinostat with chemotherapy (vincristine plus irinotecan) shows effective tumor response in HB cells in vitro, as well as in innovative patient-derived spheroids and patient-derived xenograft (PDX) mouse models. This opens up new avenues for therapeutic improvement.

Full text here: www.journal-of-hepatology.eu/article/S0168-8278(24)00040-0/fulltext

EASL - The Home of Hepatology

02/05/2024

We are pleased to share an update from Vivet Therapeutics on its GATEWAY gene therapy clinical trial.

Three consecutive groups of patients will receive an increasing dose level of VTX-801. The first patient in the second, intermediate dose has been treated.

Each participant receives a single intravenous infusion of VTX-801 in their bloodstream and is then followed-up for five years to evaluate the safety and the efficacy of the gene therapy.

Dosing of the second group followed the favorable opinion of an independent Safety Committee with four Wilson disease experts who reviewed the promising data collected in the first patient group.

Dr. Michael Schilsky, Principal Investigator at the Yale clinical trial site for GATEWAY, as well as a member of WDA’s Medical Advisory Committee, commented, “Evaluation of VTX-801 as a treatment option for Wilson disease is making good progress with no detected serious adverse events in the first dose, Cohort 1 patients.”

You can read details on the update here: https://wilsondisease.org/vivet-therapeutics-wilson-disease-update-april-2024/

The GATEWAY study, which is taking place is several locations across the US, UK, Germany and Denmark, is actively enrolling. If you’d like to learn more, email patients@vivet-therapeutics.com

19/04/2024

- April 19, 2024

"Liver disease poses a significant health and socio-economic burden globally, yet it lacks adequate attention on the global political agenda...
There is low awareness about liver disease risk factors at the population level, in particular among populations with low socioeconomic status.
Most liver diseases could be prevented."
worldliverday.org

Liver disease poses a significant health and socio-economic burden worldwide, yet it lacks
adequate attention on the global political agenda. 📣

This World Liver Day, let's prioritise liver health together! 🤝 Learn more ➡ https://bit.ly/3KWNA8O.



EASL - The Home of Hepatology

14/04/2024

13/04/2024

JPGN online on Twitter:

In with cholestatic disease, monoallelic gene variants are relatively common.

Liver biochemistry normalizes over time, emphasizing the need for appropriate counseling.

https://bit.ly/3TCuwCb

13/04/2024

JPGN online on X (Twitter):

Study examines whether could be linked to the 2022 outbreak in children. Histopathology shows nonspecific inflammation and hepatic necrosis.

https://bit.ly/3TVNEdf

08/04/2024

Very interesting Pediatric Hepatology Sessions in Milan, at

Keynote lecture: ALAGILLE: A MULTIFACETED CONDITION - Binita Kamath

08/04/2024

New insights on real-world use of for . Effective & well-tolerated in broad patient contexts.

Objective Maralixibat, an ileal bile acid transporter inhibitor, is the first drug approved by the U.S. Food and Drug Administration for the treatment of cholestatic pruritus in patients aged ≥3 mon...

03/04/2024

Key Proteo Submits De Novo Application to FDA for its First Newborn Screening Kit Potential to be the first IVD for the screening of Wilson Disease, Wiskott-Aldrich Syndrome, X-linked Agammaglobulinemia and Adenosine Deaminase Deficiency Read press release.

02/04/2024

ERN RARE-LIVER Webinar Series
09.04.2024: Liver involvement in patients with Fontan-type circulation

https://rare-liver.eu/news/detail/ern-rare-liver-webinar-series-09042024-liver-involvement-in-patients-with-fontan-type-circulation/