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Acoramidis is a selective, small molecule, orally administered, near-complete (≥90%) TTR stabilizer. These findings further support the thesis that ever better increases in serum TTR lead to ever ...

Acoramidis is a selective, small molecule, orally administered, near-complete (≥90%) TTR stabilizer. These findings further support the thesis that ever better increases in serum TTR lead to ...

AccurEdit Therapeutics today announced that its internally developed in vivo gene editing therapy ART001 has been granted a Regenerative Medicine Advanced Therapy (RMAT) designation by the U.S. Food ...

The results showed sustained reductions in serum TTR levels after a single dose, with a mean reduction of 90% by Day 28 and improvements in various clinical and biomarker measures over 24 months ...

Intellia has leveraged its expertise in Crispr/Cas9 gene editing to advance a pipeline of in vivo and ex vivo therapies for diseases with high unmet medical needs. We believe Intellia's proprietary ...

0.65–0.89]; and 70–80 mm Hg diastolic BP TTR: 0.85 [95% CI, 0.80–0.90]), major adverse cardiovascular events (120–140 mm Hg SBP TTR: 0.76 [95% CI, 0.70–0.83]), and heart failure (110–130 mm Hg SBP TTR ...

Acoramidis is an oral small-molecule TTR stabiliser whose development is more than 90% complete. The ACT-EARLY trial aims to test whether prophylactic treatment of asymptomatic carriers of the variant ...

The strategy of the REST Overseas Shares Indexed TTR managed fund is Predominantly growth assets (can range between 90-100% growth assets) consisting of overseas shares. What is the APIR code of the ...

Targeting of the 3′ untranslated region of TTR mRNA in primates resulted in 90% reduction of TTR at day 14 postinfusion and >70% reduction at day 28.102 A phase I study in mild–moderate ATTR-PN ...

Last month, the Carpet Mills Arts District (CMAD) in Yonkers was transformed like never before as it hosted its ...