Neurological efficacy and safety of RNA therapeutics in hereditary transthyretin amyloidosis: a systematic review and meta-analysis of randomized controlled trials
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Background: Hereditary transthyretin amyloidosis (hATTR) is caused by mutations in the transthyretin (TTR) gene, which lead to the aggregation of misfolded TTR protein and amyloid accumulation in the peripheral nerves, heart, and... ...
