Article
[Therapeutic strategy for familial amyloid polyneuropathy (FAP)].
Rinsho shinkeigaku = Clinical neurology - 1 Nov 2009
Ikeda Shu-ichi
Abstract excerpt
Familial amyloid polyneuropathy (FAP) was long considered to be an incurable disease, but a new therapeutic approach was developed 15 years ago. As the liver produces most of the transthyretin (TTR) in serum, it was assumed that the replacement of a liver expressing an abnormal TTR gene should stop the production of the variant TTR, the serum amyloid precursor in FAP. Until now about 1,500 FAP patients underwent...
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