Gene therapy delivered early can help children with rare neurodegenerative disease retain motor and cognitive functions

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 origin which leads to the progressive loss of the ability to walk, talk and interact. This was confirmed by a study published in the New England Journal of Medicine which showed that the therapy, if administered early, is able to preserve motor function and cognitive abilities in most patients.