Share on Facebook. Opens in a new tab or window Share on Bluesky. Opens in a new tab or window Share on X. Opens in a new tab or window Share on LinkedIn. Opens in a new tab or window The FDA approved ...

It is part of a national study which aims to identify disorders earlier.

Finding showed treatment with Lenmeldy significantly extended severe motor impairment-free survival in children with PSLI MLD compared with untreated natural history children. The Food and Drug ...

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 ...

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 ...

A genetic medicine at the heart of a $387 million Kyowa Kirin acquisition has won FDA approval, a regulatory decision that makes the one-time treatment the first approved therapy for the rare ...

CINCINNATI (WKRC) - There is promising new research for a genetic condition, which impacts thousands of families worldwide. A small company in the U.K. is giving one mother and son reason to hope. The ...

The Food and Drug Administration on Monday approved a gene therapy for metachromatic leukodystrophy, a devastating genetic disorder that eats away at affected children’s neurons. The medicine, ...

Currently, there are no approved treatments for MLD, a lysosomal storage disease caused by a deficiency in arylsulfatase A. The following article features coverage from the 17th Annual WORLDSymposium ...

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 ...