Study	Reason for exclusion
Adams 2015	A report on a phase 2 open‐label extension study of patisiran. Not randomised
Barroso 2017	A paper reporting interim results of an open‐label, single‐treatment study investigating safety and efficacy of tafamidis in TTR‐FAP. Not randomised
Coelho 2013	A 12‐month, open‐label, multicentre, international, single‐arm trial (ClinicalTrial.gov, NCT00791492) evaluating the safety and efficacy of tafamidis in people with Val30Met TTR‐FAP who completed the pivotal trial of tafamidis continues to receive tafamidis or are switched from placebo 20 mg once daily for 12 months. Not randomised
Gillmore 2015	A report on a phase 2, open‐label extension study of revusiran, an investigational RNAi therapeutic for the treatment of people with transthyretin cardiac amyloidosis. We excluded this study as it is not randomised and does not focus on FAP (neuropathy).
Gundapaneni 2018	A paper reporting a post hoc analysis comparing data from the pivotal trial of tafamidis (NCT00409175; Coelho 2012) and an open‐label study to evaluate TTR stabilisation, safety, tolerability, and efficacy of tafamidis in people with non‐Val30Met TTR‐FAP (NCT00630864; Merlini 2013). Not randomised
Maurer 2018	A paper reporting on a multicentre, international, randomised double‐blind, placebo‐controlled, phase 3 trial of tafamidis in people with transthyretin amyloid cardiomyopathy. We excluded this study as it does not focus on FAP (neuropathy).
Plante‐Bordeneuve 2018	A report mentioning an ongoing, phase 3, open‐label extension study (ClinicalTrial.gov, NCT02175004) designed to evaluate long‐term efficacy and safety of inotersen in people with TTR‐FAP who were enrolled and completed the pivotal RCT of inotersen up to 5 years. Not randomised
Russo 2013	A conference abstract presenting a small study investigating the efficacy of tafamidis in 7 people with TTR‐FAP. Not randomised
Sah 2011	A conference abstract which presents an update on the development of ALN‐TTR01 as a therapeutic option for the treatment of TTR‐related amyloidosis as well as of a second‐generation RNAi therapeutic targeting TTR. Not a trial
Suhr 2015	A paper reporting a multicentre, international, open‐label, multiple‐dose escalation phase 2 study (Clinicaltrials.gov, NCT01617967) which evaluates the safety, tolerability, pharmacokinetics, and pharmacodynamics of multiple doses of patisiran (ALN‐TTR02) in people with FAP. Not randomised
Waddington‐Cruz 2016	A paper reporting interim results from an open‐label extension study (ClinicalTrials.gov, NCT00925002) which evaluates the long‐term safety and efficacy of tafamidis in people with Val30Met TTR‐FAP over a 10‐year period. Not randomised

FAP: familial amyloid polyneuropathy; RCT: randomised controlled trial; RNAi: ribonucleic acid inhibitor; TTR: transthyretin; TTR‐FAP: transthyretin‐familial amyloid polyneuropathy.