Are we creating a new phenotype? Physiological barriers and ethical considerations in the treatment of hereditary transthyretin-amyloidosis

Abstract Hereditary transthyretin (TTR) amyloidosis (ATTRv) is an autosomal dominant, systemic disease transmitted by amyloidogenic mutations in the TTR gene. To prevent the otherwise fatal disease course, TTR stabilizers and mRNA silencing antisense drugs are currently approved treatment options. W...

Full description

Saved in:
Bibliographic Details
Main Authors: Maike F. Dohrn, Jessica Medina, Karmele R. Olaciregui Dague, Ernst Hund
Format: article
Language:EN
Published: BMC 2021
Subjects:
Online Access:https://doaj.org/article/9fb44f289ffd423bb7e7aa14a4bebefc
Tags: Add Tag
No Tags, Be the first to tag this record!