Treatment Updates for Neuropathy in Hereditary Transthyretin Amyloidosis

Purpose of review To present an overview of current and upcoming therapies for hereditary transthyretin-mediated amyloidosis with peripheral neuropathy. Recent findings. Hereditary transthyretin-mediated amyloidosis (hATTR) also known as ATTRv (v for variant) is a rare, progressively debilitating disease associated with high morbidity. The past decades have seen the development of TTR stabilizers including diflunisal and tafamidis, as well as gene silencers including inotersen, patisiran, and vutrisiran, the lattermost gaining FDA approval most recently in 2022. There are also promising therapies under investigation including gene therapy with CRISPR. Symptomatic treatment for neurologic symptoms of ATTRv are also available. Summary Traditionally a disease without available disease-modifying therapies, hATTR now has several available treatments as well as a plethora of novel modalities under investigation. There are also effective symptomatic treatments with which clinicians should become familiar.