Systemic light chain and transthyretin amyloidosis—treatment advancements and future directions
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G Bianchi, V Sanchorawala, A Wechalekar, M Grogan…
The Lancet, 2026
thelancet.com
Once rapidly fatal, neglected, and orphan diseases without approved therapeutic options, systemic amyloidoses are now highly treatable. Basic scientific discoveries regarding the molecular mechanisms of transthyretin misfolding and aggregation have driven the development of drugs for the treatment of transthyretin (ATTR) amyloidosis, with six novel therapies approved since 2018. The combination of daratumumab, cyclophosphamide, bortezomib, and dexamethasone was approved for the treatment of light chain (AL) …
