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ATTR Gene Therapy Shows Durable Benefit Despite Safety Concerns
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ATTR Gene Therapy Shows Durable Benefit Despite Safety Concerns
Nexiguran ziclumeran (nex-z), an investigational CRISPR-Cas9 gene therapy for transthyretin amyloid cardiomyopathy (ATTR-CM), continues to demonstrate long-term efficacy in early-phase research, even as its phase III trials remain on hold due to a serious safety event, as reported on Med Page Today. Key Findings from Phase I: In a 36-patient cohort, serum transthyretin (TTR) levels […]
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