Amyloidosis is a disease that can fatal, causing nerve and organ damage in those with the disease. The condition has many different forms, but one form know as transthyretin-mediated amyloidosis is hereditary. In patients with this form of the disease a protein called transthyretin builds up in the body, causing damage to organs and nerves.
For patients in England with this form of the disease there is new hope. A drug to treat it, called Patisiran has just been approved for patients by the National Health Service (NHS). Patisiran works by ‘silencing’ the faulty transthyretin gene in these patients, which prevents the toxic build up of the protein.
This gene silencing medication can either stop, or reverse, the disease which is incredible for those diagnosed with this form of amyloidosis. It also has the potential to pave the way for other gene silencing medications that could improve the lives of patients with diseases like Huntington’s and porphyria.
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