Crigler-Najjar syndrome: Gene therapy maintains its efficacy four years after treatment
The New England Journal of Medicine has published a study on the long-term efficacy of AAV8-mediated gene therapy (GNT0003) in patients with Crigler-Najjar syndrome, an extremely rare congenital liver disorder affecting approximately 500 people in Europe. It is caused by a deficiency of UGT1A1, an e
<p>The New England Journal of Medicine has published a study on the long-term efficacy of AAV8-mediated gene therapy (GNT0003) in patients with Crigler-Najjar syndrome, an extremely rare congenital liver disorder affecting approximately 500 people in Europe. It is caused by a deficiency of UGT1A1, an e</p>