Please provide your email address to receive an email when new articles are posted on . Metachromatic leukodystrophy is an incurable rare genetic disease. At 5 years of age, 71% of children given ...

Some $2 million in the governor's supplemental budget proposal could help save lives. Last month, WBAL-TV 11 News introduced ...

The FDA approved atidarsagene autotemcel (arsa-cel; Lenmeldy), the first gene therapy to treat children with pre-symptomatic late infantile, pre-symptomatic early juvenile, or early symptomatic early ...

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 ...

NORTHRIDGE, LOS ANGELES (KABC) -- Amelia and Ella have a special bond. When Ella met Amelia in kindergarten, the fact that she was different never mattered to her. Ella just wanted to help her friend.

BOISE, Idaho — Piper Morse and her son Grayson have been dreaming of a special place where they could get outside and enjoy time together. Make-a-Wish Idaho was able to step in and make that dream a ...

BOSTON and LONDON, Nov. 19, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the U.S. Food and Drug Administration (FDA) has cleared the ...