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Petitioning FDA

Approve the drug Tafamidis

In February 2012, Martin McGarry was diagnosed with Familial Amyloidosis, an extremely rare and fatal hereditary disease, which claimed the life of his mother and two brothers. In Familial Amyloidosis, the liver produces misfolded protein, which begin to attack other organs. This disease significantly diminishes the quality of life due to the symptoms including polyneuropathy, characterized by sensory loss, pain and weakness in the lower limbs, cardiomyopathy (deterioration of the heart), and severe impairment of the autonomic nervous system - causing gastrointestinal issue and unintentional weight loss ... just to name a few. As the disease progresses, patients often lose the ability to walk, needing wheelchair assistance, and eventually become bedridden and dependent on caregivers. Martin has begun to experience these symptoms, but has maintained his optimism and hope for a cure. He has been in contact with doctors who will prescribe a new drug that is not available in the United States, but the cost is extremely expensive and will not be covered by insurance. Time is of the essence for him and it is vital that he begins this drug treatment as soon as possible to the stop the progression of Amyloidosis.

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