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Life-Saving Treatment For Rare Disease Affecting Babies:NHS - 0 views

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    The new deal struck by the NHS will enable provision of a life-saving treatment for babies and young children who suffer with a rare and fatal genetic disease, metachromatic leukodystrophy (MLD). The revolutionary gene therapy treatment, known by its brand name Libmeldy, is used to treat MLD, which causes severe damage to the child's nervous system and organs, leading to a life expectancy of just five to eight years. Having a reported list price of more than £2.8 million, it is the most expensive drug in the world, but can now be offered to young patients on the NHS in England after the health service negotiated a significant confidential discount. the drug works by removing the patient's stem cells and replacing the faulty gene that causes MLD before then re-injecting the treated cells into the patient. The most common form of MLD usually develops in babies younger than 30 months and can cause loss of sight, speech and hearing, as well as difficulty moving, brain impairment, seizures, and eventually death.
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