If administered early, gene therapy has the potential to change the medical history of children born with metachromatic leukodystrophy (MLD), a rare and lethal neurodegenerative disease of genetic ...

Gene therapy, if administered early, preserves motor and cognitive functions. Newborn screening is crucial but not yet widespread. The study is the result of 20 years of research at the San ...

"If Lily had been screened at birth, we could have gone on to have treatment in six to eight weeks and prevented everything we're going through now. "To know that is devastating - I never want another ...

Advocates fear the U.S. could return to a patchwork system in which richer states provide more comprehensive screenings than ...

RFK Jr.'s Health Department shut down an advisory group that recommends how to test newborns for genetic disorders ...

The potential for cell and gene therapies is growing, offering groundbreaking treatments for rare genetic disorders and ...

"Moreover, the duration of follow-up is among the longest reported for a lentiviral vector-based gene therapy; 13 patients ...