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A clinical trial using an existing, safe, medication in a new way to help patients suffering from Marfan Syndrome. If the trial is successful, it may provide a cure for this disease within 5 years.

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Categories

  • Health/Wellbeing Health/​Wellbeing
  • Medical Research Medical Research
  • Beneficiaries

    • Women & Girls Women & Girls
    • Other Other

    Situation

    Marfan Syndrome is an inherited genetic disease which affects as many people in this country as Cystic Fibrosis. It causes swellings (aneurysms) of the aorta which can rupture with an 80% chance of death. The average age of death, despite modern treatment both medical and surgical, is still only 44 years. An exciting drug trial promises to find a cure for this disease within five years. The aim of our project is to increase the life span of Marfan Syndrome patients into the normal life span range, by protecting the aortic wall from developing aneurysms. The project has been approved by scientific peer review, and we have 500 patients age 18-40 in the UK, who have already volunteered for this study and are very eager to get started. The trial is a new use for an existing medicine shown to double the life span of the Marfan Syndrome mouse. It is a safe medication, and is widely used already for high blood pressure. A promise of £1.5 million has been made from a major foundation if we can raise a further £500,000.

    Solution

    100%
    Categories

  • Health/Wellbeing Health/​Wellbeing
  • Medical Research Medical Research
  • Beneficiaries

    • Women & Girls Women & Girls
    • Other Other