The medication tafamidis has been approved for the treatment of transthyretin<unk>  in Europe. Studies have found that it delays neurological problems when started early. The US Food and Drug Administration's Peripheral and Central Nervous System Drugs Advisory Committee rejected the drug in June 2012, in a 13-4 vote. The committee stated that there was not enough evidence supporting efficacy of the drug, and requested additional clinical trials. In August 2018, the FDA approved patisiran, an siRNA-based treatment, at an expected cost of up to $450,000 per year. In August 2021 six patients with hereditary ATTR<unk> osis with<unk>  were given doses of NTLA-2001, based on a CRISPR gene editing system. Researchers reported mild adverse events and decreases in serum misfolded transthyretin protein concentrations through targeted knockout.
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