Metachromatic leukodystrophy is a rare genetic disorder that affects between 1 and 9 people out of 100,000 and manifests itself mainly in childhood and adolescence—with 10 to 20% of cases ...

a childhood neurodegenerative disease called Globoid cell leukodystrophy (GLD), aka Krabbe disease. The approach even extended survival, according to researchers led by Chris Bennett at the University ...

DUBLIN--(BUSINESS WIRE)--Research and Markets has announced the addition of the "Globoid Cell Leukodystrophy (Krabbe Disease) - Pipeline Review, H2 2016" report to their offering. Globoid Cell ...

Also known as globoid cell leukodystrophy, Krabbe disease is classified ... In Krabbe disease, the lack of an enzyme in the lysosome causes a toxic buildup that leads to the destruction of myelin.

It’s a new gene therapy for a rare, inherited disorder called metachromatic leukodystrophy (MLD ... copy of the mutated gene that produces toxic substances that build up in their brain, kidneys ...