metachromatic leukodystrophy
Children in England with rare life-threatening illness to get world’s most expensive drug
Children with a rare and fatal genetic condition in England will soon be treated with the world’s “most expensive drug”. The NHS has struck a deal for life-saving treatment, according to a statement from the health service’s chief executive. The Libmeldy gene therapy is used to treat metachromatic leukodystrophy (MLD), a disease that causes severe […]
3 years agoTOPMOST STORY NOW

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