Each form of the disease has different symptoms. The particular type of metachromatic leukodystrophy a patient has is almost ...

What Is White Matter Disease? White matter disease is the wearing away of tissue in the largest and deepest part of your brain. The loss of white matter function has a number of causes ...

Orchard Therapeutics' gene therapy for rare childhood disease metachromatic leukodystrophy (MLD) Libmeldy has been rejected for NHS use by NICE in draft guidance. The agency said that while there ...

specifically the lysosomal storage diseases such as metachromatic leukodystrophy, and Canavan disease, both of which result from mutations in enzymes in the sulphatide pathway. There are no cures ...

In a groundbreaking development, hundreds of newborns have started being tested for over 200 rare genetic conditions as part ...

a good fit for the rare disease drugs from GSK. Aside from Strimvelis, Orchard gets two late-stage clinical programmes in ongoing registrational studies for metachromatic leukodystrophy (MLD ...

The missense mutations in both families fulfill the 'disease causing' criteria as previously described 12. It is unlikely that they represent a common polymorphism because their presence was ruled ...

CHELMSFORD-based Barratt and David Wilson Homes has successfully raised over £40,000 for its Charities of the Year, H-ABC ...