RNAi therapeutics represent a promising advancement in the treatment of ATTR-CM, offering significant benefits over placebo. It demonstrated substantial improvements in survival, functional capacity, and quality of life, alongside a marked reduction in BNP levels and GLS-critical biomarkers of disease progression. These findings underscore the potential of RNAi therapy to alter the disease trajectory and improve patient outcomes.
Keywords: AATR‐CM; RNA interference therapeutics; RNAi therapy; cardiomyopathy; transthyretin amyloidosis.
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