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- Patisiran Trial Results Reinforce RNA-Based Therapy in Rare Neuropathies

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Medical Article
Introduction Hereditary variant transthyretin amyloidosis (ATTRv) is a rare, autosomal dominant disorder marked by progressive deposition of misfolded transthyretin (TTR) protein fibrils in multiple organs. Typically presenting around age 68, it can occur earlier and is associated with a survival span of 5–15 years post-diagnosis. While oral stabil

Patisiran Trial Results Reinforce RNA-Based Therapy in Rare Neuropathies
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