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The information and materials accessed through or made available for use on any of our Sites, including, any information about diseases, conditions, treatments, or medicines, are for informational purposes only. The Content is not intended to be and is not a substitute for professional medical advice, diagnosis, or treatment, and your participation on our Sites does not create a healthcare professional-patient relationship. You should consult a doctor or other qualified health care professional regarding any questions you have about your health or before making any decisions related to your health or wellness. Call your doctor or 911 immediately if you think you may have a medical emergency.message sent
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He Inherited A Devastating Disease. A CRISPR Gene-Editing Breakthrough Stopped It
Patrick Doherty had always been very active. He trekked the Himalayas and hiked trails in Spain. But about a year and a half ago, he noticed pins and needles in his fingers and toes. His feet got cold. And then he started getting out of breath any time he walked his dog up the hills of County Donegal in Ireland where he lives. “I noticed on some of the larger hill climbs I was getting a bit breathless,” says Doherty, 65. “So I realized something was wrong.”
Doherty found out he had a rare, but devastating inherited disease – known as transthyretin amyloidosis – that had killed his father. A misshapen protein was building up in his body, destroying important tissues, such as nerves in his hands and feet and his heart.
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The symptoms of Amyloidosis and the treatments that are available vary depending on which type of Amyloidosis a patient has been diagnosed with. Select which type you want to learn more about, and oneAMYLOIDOSISvoice can highlight the resources that are most relevant to your MPS/ML type.
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