UF scientist develops safer gene therapy restoring vision in children with rare inherited eye diseases; pivotal trials begin ...
A first-in-human clinical trial evaluating an investigational gene therapy targeting complement-mediated pathways in ...
Scott Pelley, one of the most experienced and awarded journalists today, has been reporting stories for 60 Minutes since 2004. The 2024-25 season is his 21st on the broadcast. Scott has won half of ...
A groundbreaking gene therapy trial has successfully restored hearing in individuals with a rare form of congenital deafness.
The FDA has approved Fondazione Telethon ETS’s etuvetidigene autotemcel (Waskyra) for Wiskott–Aldrich syndrome (WAS). Etuvetidigene autotemcel is the first gene therapy to be approved for this rare ...
A new study has brought scientists one step further in the direction of developing a cure for a brutal group of rare brain disorders known as SYNGAP1-related disorders, or SRDs. Researchers were able ...
Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...
Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall Street, and biotechnology. He is also a co-host of the weekly biotech podcast ...
Forbes contributors publish independent expert analyses and insights. A patient in a late-stage gene editing trial for a rare heart condition died from fatal liver complications after receiving an ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results