Leukodystrophy refers to a group of rare genetic disorders affecting the central nervous system (CNS). The CNS includes the brain and spinal cord. The outlook for people with these disorders is ...

Leukodystrophy is a group of rare genetic neurological diseases that cause the sufferer to revert to a child-like state. In a Channel 4 documentary, The Curious Case of the Clark Brothers, living with ...

In metachromatic leukodystrophy (MLD), the deficiency of the lysosomal enzyme arylsulfatase A (ARSA) leads to demyelination in the central and peripheral nervous system and ultimately to death.

Montréal researchers discover a new crucial gene associated with these orphan diseases Leukodystrophies are deadly neurodegenerative diseases that affect one in 7,000 children and remain incurable.

A team from the McGill University Health Centre Research Institute has developed the first representative animal model of 4H leukodystrophy, a common form of the fatal and currently incurable ...

A scientist in a race against time to find a cure for her daughter's rare and deadly disease has said they are "finally in the driving seat". Dr Michelle Teng is trying to develop the world's first ...

A 3 year-old girl presented with progressive spasticity, visual loss and loss of motor milestones. Metachromatic Leukodystrophy (MLD): Flair axial MRI images. Note the bilateral confluent white matter ...

Orchard Therapeutics has secured reimbursement from another European market for Libmeldy, an ex vivo gene therapy for the rare childhood disease metachromatic leukodystrophy (MLD) first approved in ...