Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study

In this interim 12-month analysis (n=211) of an ongoing global study, patisiran, a systemically delivered RNA interference therapy, maintained efficacy with an acceptable safety profile; improvements in polyneuropathy (modified Neuropathy Impairment Score +7) were sustained.

Source:

The Lancet Neurology