Non-alcoholic fatty liver disease (NAFLD) is a hepatic metabolic syndrome resulting from lipid metabolic imbalance.

A new study in Human Gene Therapy describes a machine learning (ML) model that can be used as a surrogate for laborious in ...

the development of an AAV-based therapy that would target the liver specifically was a key advantage of using the AAV platform. A number of AAV vectors have been engineered using different viral ...

A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy ...

This story was originally published on PharmaVoice. To receive daily news and insights, subscribe to our free daily PharmaVoice newsletter. The death of a 16-year-old boy taking a gene therapy ...

Acute liver injury is a known possible side effect of Elevidys and other AAV-mediated gene therapies and is highlighted in the prescribing information. Although it is not a new safety signal and ...

The two deaths occurred in children in Russia and Kazakhstan, with the liver failure starting five to six weeks after administration of the adeno-associated virus-based treatment. Both children ...

In both studies, adeno-associated virus 2 (AAV2) was detected in cases that required transplants as well as the vast majority of hospitalized cases, but was detected in very few of the matched healthy ...

Following the safety update on acute liver failure that was issued on ... is a single-dose, adeno-associated virus (AAV)-based gene transfer therapy for intravenous infusion designed to address ...

and some research has suggested systemic administration of AAV can cause damage to liver cells and inflammation. Astellas voluntarily suspended the study after the fourth patient became ill ...

Australia: In a significant development for Hemophilia B treatment, a multi-year follow-up study has shown that a single ...