Preclinical and phase I data for $AKCA-TTR-LRx (second generation inotersen, ASO conjugated with GalNAc), currently being tested in CARDIO-TTRansform, the largest #ATTR#Amyloidosis trial to date. Tafamidis allowed without restrictions.
There are many new exciting trials in transthyretin #amyloidosis – as we continue to enroll patients in the CARDIO-TTRansform trial, I thought I would highlight some of these trials in ATTR-CM
If you recall, 2 main approaches to treating ATTR #amyloidosis with approved drugs (figure credit @frederickruberg@maz_hanna )
1)Stabilizers: bind to the TTR tetramer to stabilize it. #tafamidis , AG-10, diflunisal
2)Silencers: Inotersen and Patisiran
tafamidis is prescribed to ATTR-CM pts & inotersen/patisiran for those with hATTR polyneuropathy
But – 2 main questions in ATTR-CM
1)Which is associated with better outcomes – silencer or stabilizer?
2)Is the combination of silencer+stabilizer superior to stabilizer only?
Do you know the name of this sign on #echofirst?
#Cardiotwitter #ACCFIT
In 2006, Pieroni et al called this the "Binary Sign". They identified this myocardium appearance in 40 patients with Fabry's disease. When compared to patients with HCM, HTN, and healthy subjects, the sign had 94% sensitivity and 100% specificity. ( )ncbi.nlm.nih.gov/pubmed/16631007
Hypothesis: endomyocardial glycosphingolipids compartmentalization: 1) thickened glycolipid-rich endocardium 2)free glycosphingolipid subendocardial storage 3)an inner severely affected myocardial layer with a clear subendocardial-midwall layer gradient of disease severity.