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In a previous Phase I trial, six patients treated with a single dose of NTLA-2001 showed significant reductions in serum TTR protein levels, with no serious adverse effects or liver damage reported

It affects both a persons mood and their ability to function and is associated with increased risk of suicide (Cavanagh et al

There was a significant reduction in ulcer size in all groups (p G pathogenic variant) in the first year of life, based in the presence of hyperphagia, hepatomegaly, prominent muscles in the limbs and diabetes mellitus (A1c 12,9%)

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