New therapeutic approaches for familial amyloidotic polyneuropathy (FAP).

Abstract excerpt

Because the use of liver transplantation as therapy for familial amyloidotic polyneuropathy (FAP) has given rise to several problems, an alternative treatment is needed. We have tried several other approaches. To suppress production of variant transthyretin (TTR) in the liver, we injected purified normal human TTR into patients with FAP. Production of variant TTR decreased significantly for 48 h. However, 1 week...

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