A designer drug for amyloid polyneuropathy

摘要

There are frustratingly few specific treatments available for the 100-odd etiologies of polyneuropathy. Some notable exceptions aside (e.g., dapsone and ri-fampin for leprosy, IV immunoglobulin for chronic inflammatory demyelinating polyradiculoneuropa-thy), symptomatic treatments, such as ankle-foot or-thoses or drugs for neuropathic pain, form the bulk of the therapeutic armamentarium for polyneuropathy. Specific treatments are even more elusive in the genetic polyneuropathies. Recent hopes from animal models that vitamin C might be effective in type 1 Charcot-Marie-Tooth disease have been unrealized.1 In this context, the report about tafamidis in familial amyloid polyneuropathy (FAP) in this issue of Neurology is of particular interest.