Although not frequent, two recent large‐scale trials showed severe adverse events under gene modifier therapy could be fatal, including cardiac failure, acute kidney injury and thrombocytopenia.33, 34 This makes safety of gene modifier therapy a concern for patients with a late onset, rapid progression, or marked cardiac dysfunction, just like the condition of A97S TTR‐FAP. The gene discussed is FAP; the disease is Thrombocytopenia.