Crigler Najjar Syndrome News and Research

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Preliminary results of AAV-mediated gene therapy for Crigler Najjar syndrome

Preliminary results from the European gene therapy trial for Crigler-Najjar syndrome, conducted by Genethon in collaboration with European network CureCN, were presented at the EASL (European Association for the Study of the Liver) annual International Liver Congress on June 26.

28 Jun 2021

Landmark 30-year study describes clinical course of patients with Crigler-Najjar syndrome

A new study summarizes more than 30 years of clinical experience and describes the clinical course of 28 individuals homozygous for damaging mutations in the UGT1A1 gene who were born between 1984 and 2015 with Crigler-Najjar syndrome.

17 Apr 2020

Researchers are developing and testing novel gene therapy to cure Crigler-Najjar syndrome

Crigler-Najjar syndrome affects about one in a million people at birth. Because of a defective gene, these individuals lack an essential enzyme in their liver to eliminate bilirubin, a toxic bile pigment that accumulates in all body tissues causing jaundice and potentially leading to irreversible and lethal neurological damage.

28 Feb 2019

Regenerative medicine scientist receives $1.3 million to study stem cell therapy for liver failure

Currently, the only therapy for metabolic liver disease is an organ transplant. Tracy Grikscheit, MD, an attending physician and regenerative medicine scientist at Children's Hospital Los Angeles, hopes to change that reality.

7 Nov 2018

Collaborative effort to develop rare disease patient registries

The Orphan Disease Center in the Perelman School of Medicine at the University of Pennsylvania will collaborate with Pulse Infoframe Inc., a medical informatics company, to develop rare disease patient registries aimed at enabling international collaboration to better understand these diseases, as well as accelerate and improve clinical studies to develop new therapies.

31 May 2017

Promethera Biosciences completes patient enrolment for first clinical trial of HepaStem

Promethera Biosciences, a Belgian biotechnology company developing Promethera HepaStem, a cell-based therapy for the treatment of liver-based metabolic diseases including Crigler-Najjar Syndrome and Urea Cycle Disorders, announces today the completion of patient enrolment for its first clinical trial.

12 Nov 2013

Urea cycle disorder patient successfully treated by Promethera(R) HepaStem in Phase I/II trial in France

Promethera Biosciences, a Belgian biotechnology company developing Promethera(R) HepaStem, a cell-based therapy for the treatment of liver-based metabolic diseases including Crigler-Najjar Syndrome and Urea Cycle Disorders, announced that it has successfully treated a patient with liver progenitor cells enrolled in its Phase I/II trial.

1 Mar 2013

Liver disease therapies: an interview with Eric Halioua, CEO of Promethera Biosciences

Liver disease is a very broad family of diseases. There are two main categories of liver disease:

27 Nov 2012

Crigler Najjar Syndrome News and Research

Preliminary results of AAV-mediated gene therapy for Crigler Najjar syndrome

Landmark 30-year study describes clinical course of patients with Crigler-Najjar syndrome

Researchers are developing and testing novel gene therapy to cure Crigler-Najjar syndrome

Regenerative medicine scientist receives $1.3 million to study stem cell therapy for liver failure

Collaborative effort to develop rare disease patient registries

Promethera Biosciences completes patient enrolment for first clinical trial of HepaStem

Urea cycle disorder patient successfully treated by Promethera(R) HepaStem in Phase I/II trial in France

Liver disease therapies: an interview with Eric Halioua, CEO of Promethera Biosciences

Expanding research and clinical options for children with cancer

Examining how pain could play a direct protective role in the gut

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