In the absence of a liver transplant,<unk>  is invariably fatal, usually within a decade. The disadvantage of liver transplantation is that approximately 10% of the subjects die from the procedure or complications resulting from the procedure, which is a form of gene therapy wherein the liver expressing wild type and mutant TTR is replaced by a liver only expressing wild type TTR. Moreover, transplanted patients must take immune suppressants (medications) for the remainder of their life, which can lead to additional complications. In late 2011, the European Medicines Agency approved the transthyretin kinetic stabilizer Tafamidis or Vyndaqel discovered by Jeffery W. Kelly and developed by FoldRx pharmaceuticals (acquired by Pfizer in 2010) for the treatment of<unk>  based on clinical trial data. Tafamidis (20 mg once daily) slowed the progression of<unk>  over a 36-month period and importantly reversed the weight loss and muscle wasting associated with disease progression.
Familial amyloid polyneuropathy
Familial amyloid neuropathy
MGUS polyneuropathy
Distal hereditary motor neuropathy type V
Muscular dystrophy
Charcot–Marie–Tooth disease
Hereditary sensory and autonomic neuropathy type I
Hereditary inclusion body myopathy