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 ...

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

For most parents, newborn screening tests are a barely perceptible safety net. But for the small minority whose babies get ...

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

Karla Wolford reaches out to hug her son, Kasyn, as she exits a plane with her daughter, Jalyn, on Wednesday, April 9, 2025, at the Fargo Jet Center. Jalyn received a bone marrow transplant in ...

The Moorhead High senior has 4H Leukodystrophy, a rare, genetic, progressive neurological condition. A benefit for Cullen is set for this Saturday at the TAK in Dilworth.

A federal advisory committee that provides guidance on which rare diseases newborns should be screened for has been dissolved by the Trump administration, according to a letter obtained by the Boston ...

Kids with Metachromatic Leukodystrophy, a rare genetic disease, often don’t even know they have it until they suddenly lose the ability to walk and talk, slowly go blind, lose control of all ...

"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 ...