World's 'most expensive' drug recommended for NHS use
Joe and Connie Elson: Around four babies are born with the disorder in England every year
"It was not long after that, that we got the diagnosis of MLD," she said. "It was completely out of the blue, there was nothing like this in my family or my husband's family." MLD, or Metachronatic Leukodystrophy, is an extremely rare hereditary disorder caused by a crucial enzyme deficiency. Over time the nerves in the brain and other parts of the body stop working properly.
The long-term prognosis for children given the drug is still unknown, and Joe will need to be monitored for years to come, but both doctors and families are hopeful it could offer a permanent cure for the condition.Libmeldy is a so-called orphan drug - a pharmaceutical agent developed to treat a medical condition which, because it is so rare, may not be profitable to produce without some form of government assistance.
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