Parents of boys with Duchenne muscular dystrophy weigh in on drug innovation and medical regulation.

Currently, GenAns Biotech is conducting investigator-initiated trials for several gene therapies, including GA001 for ...

CGT Global, a leading biotechnology company driving the development and delivery of innovative cell and gene therapies, today ...

The primary safety risks associated with gene therapy include immune reactions and off-target effects in unintended organs, ...

Sarepta Therapeutics (SRPT) stock in focus as report says company hired a Trump-linked lobbying firm after deaths linked to ...

US Food and Drug Administration Commissioner Marty Makary said he’s trying to persuade Vinay Prasad, the agency’s former head ...

A 26-year-old Downriver man seen at Children’s Hospital of Michigan in Detroit since infancy for a rare bleeding disorder in ...

Gene therapy appears to be a promising approach for a subset of genetic deafness, although challenges remain with development ...

The FDA's approval of the first CRISPR-Cas9–based gene therapy marked a major milestone in biomedicine, validating genome ...

Sarepta did not hold an investor call for its second-quarter earnings report or provide an updated full-year revenue outlook.

Connecticut is now participating in a new federal program aimed at expanding treatment access for patients with sickle cell ...

A man born with Usher syndrome type 1b, a rare genetic disease that causes congenital deafness and progressive blindness, has ...