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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.

The drug, alpelisib, which is the 100th cancer drug that has being fast-tracked to patients under the NHS Cancer Drugs Fund (CDF) will be used in combination with the hormone therapy, fulvestrant, to target the gene that causes fast-growing tumours. Up to 3,000 people a year with a certain type of secondary breast cancer will benefit from the treatment. The approach has contributed to people in England having access to nearly one third more cancer drugs compared to the European average. The drug which is manufactured by pharmaceutical company Novartis, is part of a growing number of precision treatments that target a tumour based on mutations in its DNA and that the NHS is rolling out. John Stewart, NHS National Director for Specialised Commissioning said, "In just over five years, more than 80,000 people have benefitted from earlier access to a range of cancer drugs, with people in England having access to nearly one third more cancer drugs compared to the European average, and this latest innovative new treatment will help up to 3,000 more to live a better quality of life.