transthyretin

- The primary endpoint was met (Win Ratio of 1.8) with a highly statistically significant p-value (p<0.0001); this primary endpoint result consistently favored acoramidis treatment…

PALO ALTO, Calif., July 14, 2023 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio), a commercial-stage biopharmaceutical company focused on genetic diseases and…

Serum TTR reductions were sustained at all doses tested with follow-up now reaching 12 months in the 0.1 and 0.3 mg/kg and six months in the 0.7 and 1.0 mg/kg cohorts Pharmacokinetic…

- NT-proBNP, a biomarker of cardiac failure and independent predictor of mortality in ATTR-CM patients, was stable or improving throughout the study. At Month 30, median change from…

Achieved 86% and 93% mean serum TTR reduction by day 28 at 0.7 mg/kg and 1.0 mg/kg doses, respectively, with dose-dependent reductions observed across all four dose levelsDurable serum TTR…

An intravenous CRISPR infusion lowered levels of a disease-causing protein in vivo for the…