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

Abstract excerpt

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. With 90% of the amyloidogenic protein produced by the liver, disease progression including polyneuropathy and cardiomyopathy,...

Topics

Share this publication in a Topic to start or enrich a Post.

• Share to Amyloidosis: Diagnosis, Treatment, Outcomes
• Share to Chromatin Remodeling and Cancer
• Share to Neuroendocrine Tumor Research Advances