Hereditary amyloidogenic transthyretin (ATTRv) amyloidosis with polyneuropathy (also
known as familial amyloid polyneuropathy) is a condition with adult onset caused by …

Amyloid transthyretin (ATTR) amyloidosis with polyneuropathy (PN) is a progressive,
debilitating, systemic disease wherein transthyretin protein misfolds to form amyloid, which …

The field of gene therapy is striving more than ever to define a path to the clinic and the
market. Twenty gene therapy products have already been approved and over two thousand …

Compounds containing an Ssingle bondF bond have garnered intense interest in the
chemical and biological literature. In particular, sulfonyl fluorides (RSO 2 F) are commonly …

Hereditary transthyretin amyloidosis (hATTR) with polyneuropathy (formerly known as
Familial Amyloid Polyneuropathy) is a rare disease due to mutations in the gene encoding …

Ten years after Fire and Melo's Nobel Prize for discovery of gene silencing by double-
stranded RNA, a remarkable progress was achieved in RNA interference (RNAi). Changes …

Introduction: Hereditary transthyretin-mediated amyloidosis is caused by a mutation in
transthyretin (TTR) gene resulting in misfolded TTR protein accumulating as amyloid fibrils …

Objective To assess the natural history and treatment effect on survival among patients with
transthyretin-associated familial amyloid polyneuropathy (TTR-FAP) stage 1 Val30Met …

Heritable neurological disorders provide insights into disease mechanisms that permit
development of novel therapeutic approaches including antisense oligonucleotides, RNA …

Systemic amyloidosis can be hereditary or acquired with autosomal dominant mutations in
the transthyretin gene (TTR) being the most common cause of hereditary amyloidosis …