Transthyretin Amyloid Cardiomyopathy
FDA approves vutrisiran for ATTR amyloidosis with cardiomyopathy
Alnylam announced the FDA approval of its supplemental new drug application for vutrisiran, an RNA interfering small molecule transthyretin stabilizer, for the treatment of transthyretin amyloidosis with cardiomyopathy.
Acoramidis may improve cardiac mass, function in transthyretin amyloid cardiomyopathy
ATLANTA — Data from a small substudy with cardiac MRI suggest acoramidis may improve both cardiac mass and function for adults with symptomatic transthyretin amyloid cardiomyopathy, building on previous positive data, researchers reported.
‘Across the board’ benefits seen with acoramidis for transthyretin amyloid cardiomyopathy
Acoramidis, an investigational treatment for symptomatic transthyretin amyloid cardiomyopathy, was associated with marked improvements in all-cause mortality and CV-related hospitalizations compared with placebo, data show.
Phase 3 data show acoramidis improves outcomes in transthyretin amyloid cardiomyopathy
Acoramidis, a treatment for symptomatic transthyretin amyloid cardiomyopathy, was associated with marked improvements in all-cause mortality and CV-related hospitalizations compared with placebo, according to a press release from BridgeBio.
APOLLO-B
Evaluating efficacy and safety of patisiran (Onpattro, Alnylam) in adults with transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy.
CRISPR-based genome editing therapy may help in ATTR amyloidosis with cardiomyopathy
CHICAGO — A novel CRISPR-based in vivo gene editing therapy reduced transthyretin levels in patients with hereditary transthyretin amyloidosis with cardiomyopathy, researchers reported at the American Heart Association Scientific Sessions.
Patisiran beneficial in ATTR amyloidosis with cardiomyopathy
In patients with transthyretin-mediated amyloidosis with cardiomyopathy, patisiran improved functional capacity and quality of life at 12 months compared with placebo, according to the results of the APOLLO-B trial.
Trial of acoramidis for transthyretin amyloid cardiomyopathy misses primary endpoint
In the 12-month topline results of the ATTRibute-CM trial, acoramidis, a treatment for symptomatic transthyretin amyloid cardiomyopathy, did not meet its primary endpoint, BridgeBio Pharma announced.
Tafamidis confers 5-year survival benefit in transthyretin amyloid cardiomyopathy
Compared with placebo, tafamidis was linked to improved long-term survival in patients with transthyretin amyloid cardiomyopathy, according to new data from the ATTR-ACT trial.
Tafamidis stabilizes transthyretin in cohort with transthyretin amyloid cardiomyopathy
According to a non-trial, published in JACC: CardioOncology, in unselected patients, serum transthyretin change after tafamidis initiation for transthyretin amyloid cardiomyopathy may serve as a surrogate measure of drug efficacy.