Marfan Trust

Medication in Marfan syndrome

Urgent funding is required to support a national trial of 500 patients, with a serious life threatening inherited heart disease (Marfan syndrome) using a medication which is known to strength blood vessels.

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Registered Charity in England and Wales (328070)

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    Category

  • Health/WellbeingHealth/Wellbeing

    Helping

  • Children (3-18)Children (3-18)
  • Older PeopleOlder People
  • Women & GirlsWomen & Girls
  • Young People (18-30)Young People (18-30)
  • OtherOther

Location

Situation

Recent evidence suggests that a commonly used blood pressure drug may prevent enlargement and tearing of the aorta (the main artery in the heart), which causes early death for people with the common inherited heart disease, Marfan syndrome. Modern preventive treatments have extended the average life span to 44 years, and include taking a medicine called a beta-blocker which slows the expansion of the aorta. However, this treatment will not stop the aorta expanding and tearing and eventually patients with Marfan syndrome are required to undergo major surgery to replace the enlarged aorta. At present 70% still die of acute cardiac events.

Solution