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 ...

As the May 19 legislative deadline approaches and we enter the final stretch of the 2025 session, the single most important ...

"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 ...

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.

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 ...