TTR and Nager acrofacial dysostosis: Disease-modifying therapies are currently available in CA, such as chemotherapy agents (alkylating agents, proteasome inhibitors, immunomodulatory drugs, and monoclonal antibodies targeting clonal cells), in AL amyloidosis, transthyretin stabilizers (tafamidis and acoramidis), and gene silencers (patisiran and vutrisiran), in transthyretin CA and in AFD, such as enzyme replacement therapy (ERT) (agalsidase-alpha, agalsidase-beta, and pegunigalsidase-alpha) and oral chaperone therapy (migalastat).