Acoramidis approved to reduce CV death, hospitalization for cardiac amyloidosis BridgeBio Pharma announced that acoramidis, its small molecule stabilizer of transthyretin, has been approved by the FDA for the reduction of CV death and hospitalization in patients with transthyretin amyloid cardiomyopathy....
Genes uniquely connected to depression were enriched for striated and cardiac muscle hypertrophy (HTR2B, TCAP, and TTN; Fig. 2d). Anxiety shares most of its genes with depression and has only eight unique genes (Fig. 2d). Two of these, HCN4 and ADCY8, have also been associated with ...
Discovery of a novel cardiac-specific myosin modulator using artificial intelligence-based virtual screening Modulation of the myosin molecular motor has emerged as a potential therapeutic target for both heart disease and heart failure. Here, the authors use AI-based virtual high throughput screening to...
NXC-201 has received orphan drug designation from the European Commission for use in patients with amyloid light-chain amyloidosis. The BCMA-targeted CAR T-cell therapy, NXC-201 (formerly HBI0101), has received orphan drug designation (ODD) from the European Commission for use as a po...
Moreover, MSC-secreted exosomes demonstrated the ability to improve cardiac function and reverse autophagy induced by ischemia by inhibiting the p53/Bnip3 signaling pathway. The authors propose that the existence of miR-125b-5p within these exosomes contributes to their therapeutic benefits [42]. ...
This is the second RNAi drug approved from Alnylam, after Onpattro for hereditary transthyretin-mediated amyloidosis. Alnylam also has competitors – Pfizer’s Vyndaqel treats the cardiac symptoms of amyloidosis, by binding to the transthyretin protein and slowing the formation of amyloid that causes ...
From Table 1, we have learned that amyloid may not be the only critical step or the only mechanism of action to be targeted in AD, and it may be necessary to target other mechanisms and pathways for effective treatment of AD. In December 2019, the European Union-North American Clinical Tr...
Therapeutic strategies for Alzheimer's disease. Nature Rev. Drug Discov. 1, 859–866 (2002). Article CAS Google Scholar Shearman, M. S. et al. L-685,458, an aspartyl protease transition state mimic, is a potent inhibitor of amyloid β-protein precursor γ-secretase activity. Biochemistry ...
Exosomes as critical agents of cardiac regeneration triggered by cell therapy. Stem Cell Rep. 2, 606–619 (2014). Article CAS Google Scholar Eleuteri, S. & Fierabracci, A. Insights into the secretome of mesenchymal stem cells and its potential applications. Int. J. Mol. Sci. 20, 4597 (...
While the naturally occurring or "wild type" transthyretin protein is prone to aggregate in older people causingcardiac disease, a variety of destabilizing mutations lead either to a primary cardiomyopathy or to early onset forms of polyneuropathy, known as TTR familial amyloid polyneuropathy, affecting...