Why the study?
Does a single intravenous infusion of nexiguran ziclumeran safely reduce serum TTR levels in patients with ATTR-CM?
Does a single intravenous infusion of nexiguran ziclumeran safely reduce serum TTR levels in patients with ATTR-CM?
A single dose of the CRISPR-Cas9 gene editing therapy nexiguran ziclumeran produced rapid, deep, and durable reductions in serum TTR levels over 12 months in patients with ATTR-CM, with an acceptable safety profile.
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Fontana et al. (2024) studied this question.
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