Pyrazoles are organic compounds of the general formula C3H3N2H. It is a five-membered heterocycle consisting of three carbon atoms and two adjacent nitrogen atoms. As an H-bond-donating heterocycle, pyrazole has been used as a more lipophilic and metabolically more stable bioisomer of phenol. Pyrazoles have attracted more and more attention due to their broad spectrum of action and strong efficacy.
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In 2023, BridgeBio announced positive results from ATTRibute-CM, a Phase 3 study of acoramidis in ATTR cardiomyopathy (ATTR-CM). For patients with ATTR-CM, TTR stabilization offers the chance to both preserve the cardio-protective benefits of TTR and address the root cause of disease. Acoramidis (AG10) is an investigational, next-generation, orally-administered, highly potent, small molecule stabilizer of transthyretin (TTR).
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