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 ...

And it has paved the way for other therapeutic strategies, including the first gene therapy delivered through skin grafts, which was approved this year for a form of the condition known as recessive ...

New York State Troopers spent an afternoon Thursday at Golisano Children's Hospital, engaging with children and highlighting the groundbreaking effects of gene therapy. READ MORE: <a href=" ...

A new gene therapy developed at the Children's Hospital of Philadelphia has been approved by the FDA and is being used to transform lives. The first patient to receive this gene therapy after it was ...

ST. LOUIS, Mo. (First Alert 4) - A gene therapy recently approved by the FDA to treat patients with Sickle Cell Anemia is now being administered in St. Louis. In 2023, the FDA approved Lyfgenia, ...

Thanks to in-utero blood transfusion technology, what was once a fatal diagnosis in the womb can now result in live births. However, this medical advancement created a new challenge: a growing ...

UC Davis Health researchers are testing a new gene therapy that could be transformational for patients with Angelman syndrome ...

Youth with congenital deafness treated with gene therapy showed better progress on certain hearing measures than counterparts treated with cochlear implantation in a cohort study from China. Gene ...

A new gene therapy has shown promising results in restoring hearing to children, teens, and even a young adult born with a rare form of inherited deafness. Researchers in China tested the treatment on ...

KaDee Troop is the mother of seven adopted children, four of whom have a rare genetic disorder that causes their skin to blister and tear at the slightest touch. Wounds heal slowly — if at all — and ...