Work

Tafamidis and the Paradox of Rare Disease Therapies

Year: 2024

Type: article

Abstract: Cardiac transthyretin amyloidosis (ATTR-CM) is an underrecognized cause of heart failure with preserved ejection fraction in older adults, affecting approximately 120 000 US adults. 1 It is caused by the misfolding of transthyretin, which produces insoluble amyloid fibrils that deposit in interstitial space between cardiac myocytes.2 Deposition of amyloid fibrils triggers cellular injury and reduces muscle compliance, eventually resulting in a restrictive cardiomyopathy.There are 2 types of

Source: JAMA Network Open

DOI: 10.1001/jamanetworkopen.2024.27219

Authors: Sérgio Renato da R. Decker, Dhruv S. Kazi

Institutions: Beth Israel Deaconess Medical Center, Harvard University, Hospital Moinhos de Vento

Language: English

Topic: Amyloidosis: Diagnosis, Treatment, Outcomes

Subfield: Molecular Biology

Field: Biochemistry, Genetics and Molecular Biology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: gold

Estimated APC paid: $3,000