In a phaseI clinical trial, a 22 day schedule of subcutaneous administrationof 300 mg of inotersen led to reductions of plasma TTR protein upto 76% for 4 weeks after the last dose,17 and the drug is now used in the treatment of the polyneuropathyof hereditary transthyretin-mediated amyloidosis in adults. This evidence concerns the gene TTR and amyloidosis.