The FDA on Tuesday approved etuvetidigene autotemcel (Waskyra) as the first cell-based gene therapy for patients 6 months and ...

Three years after introducing Hemgenix, the first gene therapy for hemophilia B, CSL Behring has released long-term data ...

Kids and teenagers with spinal muscular atrophy had more mobility after receiving a single-dose gene replacement therapy, ...

Steven Pipe, Professor of Pediatrics and Pathology at the University of Michigan, explained: “The five-year HOPE-B results ...

When the presenters enquired about her well-being, the brave teenager responded: "It's just incredible. You know, I've gone ...

The status could support staged transitions to new manufacturing processes, potentially mitigating some risks of high-stakes ...

BBC Breakfast viewers were moved on Tuesday when Sally Nugent and Jon Kay interviewed a guest who had received the all-clear ...

The New York Blood Center (NYBC) opened its first-ever donor center based in Queens, marking a milestone for the organization ...

Elana Thieme and Mark Mendoza, fourth-year graduate students in the Molecular and Cell Biology (MCB) PhD Program jointly ...

Initial safety results for the full cohort of 19 indicated that the treatment was well tolerated. The only notable adverse ...

Bavier joins Sudo from norovirus vaccine developer HilleVax, where he was GC and chief administrative officer. He brings more ...

Chicago's Lurie's Children's Hospital will expand care for patients living with rare genetic diseases and conditions thanks ...