Liver transplantation has proven to be effective for ATTR<unk><unk> osis due to Val30Met mutation. In 2011 the European Medicines Agency approved tafamidis for this condition. The FDA rejected the application for marketing approval in the US in 2012 on the basis that the clinical trial data did not show efficacy based on a functional endpoint, and the FDA requested further clinical trials.
Familial amyloid neuropathy
Familial amyloid polyneuropathy
Distal hereditary motor neuropathy type V
MGUS polyneuropathy
Hereditary sensory and autonomic neuropathy type I
Muscular dystrophy
Myotonic dystrophy
Canavan disease