After winning a breakthrough therapy designation for its Hunter syndrome enzyme replacement therapy, Denali Therapeutics is ...

Thirty patients have received the gene therapy within a clinical trial and early-access program, and the foundation also hopes to seek approval in the US.

In this manuscript, we give an overview of historical aspects, as well as the current state of clinical trials and approved gene therapy products for commercialization in Brazil. Focusing on gene ...

Gene therapy using modified blood stem cells helped five adults with severe hemophilia A reach therapeutic levels of clotting ...