トランスサイレチン型心アミロイドーシス（ATTR-CM）とは

トランスサイレチン型心アミロイドーシス（ATTR-CM）とは

ATTR-CMは、肝臓で作られたトランスサイレチンの突然変異型タンパク質が心臓の左心室に蓄積してアミロイドを形成してしまい、心臓の機能を損なう進行性の病気だそうです。

Transthyretin amyloidosis (ATTR-CM) occurs when your liver produces faulty transthyretin (TTR) proteins. Clumps of these abnormal proteins (called fibrils) build up in your heart’s main pumping chamber. https://my.clevelandclinic.org/health/diseases/17855-amyloidosis-attr

ATTR-CMは稀な疾患で、他の疾患に間違えられてしまうことも多いのだそう。

What You Need to Know about This Rare Heart Condition Pfizer チャンネル登録者数 7.03万人

• https://www.youtube.com/watch?v=UTW-VNFT8as　ファイザーの説明動画（4分間）

ATTR-CMの治療戦略

第1相試験の結果を報告した論文がNEJMに掲載。

1. 心アミロイドーシスに有望な抗ATTR抗体 2023年06月05日 MedicalTribune
2. Phase 1 Trial of Antibody NI006 for Depletion of Cardiac Transthyretin Amyloid　BACKGROUND Transthyretin amyloid (ATTR) cardiomyopathy is a progressive and fatal disease caused by misfolded transthyretin. Despite advances in slowing disease progression, there is no available treatment that depletes ATTR from the heart for the amelioration of cardiac dysfunction. NI006 is a recombinant human anti-ATTR antibody that was developed for the removal of ATTR by phagocytic immune cells.

Transthyretin Amyloid Cardiomyopathy (ATTR-CM)は何の略？

CMはcardiomyopathy

TTRは、transthyretin

Aはamyloid　もしくはamyloidosis

のことのようです。

Transthyretin amyloid cardiomyopathy (ATTR-CM) is one of the types of systemic amyloidosis in which misfolded transthyretin (TTR) protein gets deposited in the myocardium.

Transthyretin amyloidosis (ATTR) is a systemic disease.

Transthyretin Amyloid Cardiomyopathy (ATTR-CM) - StatPearls - NCBI Bookshelf

Transthyretin amyloid cardiomyopathy (ATTR-CM) is one of the types of systemic amyloidosis in which misfolded transthyretin (TTR) protein gets deposited in the ...

www.ncbi.nlm.nih.gov