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Morquio Syndrome: 'Without the drug, I know I don’t have long left'Children and adults living with this rare and debilitating disease are facing an uncertain future, as a life-changing drug has been denied NHS fundingOne child in 25 in the UK is born with a genetic disorder, many of them extremely rare. Here, families share their stories about living with unusual conditions Anna Eaton has two sons: Archie, 13, and Isaac, 12. Both have Morquio Syndrome, also known as MPS IVA, which affects bone development and restricts growth. It is so rare that only 105 children and young adults in the UK have it.Untreated, it leads to the progressive deterioration of mobility, health and stamina. Sufferers rarely live beyond their 20s. However, in 2008 Archie and Isaac started on a clinical trial for a new drug treatment – the first ever for this rare condition. The drug, Vimizin, replaces a missing enzyme, the cause of the disabilities and health problems, and the results – for Archie, Isaac and all the patients involved in the trial – were impressive.