Neurological efficacy and safety of RNA therapeutics in hereditary transthyretin amyloidosis: a systematic review and meta-analysis of randomized controlled trials.

ABSTRACT:

Background

Hereditary transthyretin amyloidosis (hATTR) is caused by mutations in the transthyretin (TTR) gene, which lead to the aggregation of misfolded TTR protein and amyloid accumulation in the peripheral nerves, heart, and gastrointestinal tract. Recently, RNA therapeutics, including small interfering RNAs (siRNAs) and antisense oligonucleotides (ASOs), have been approved for treating patients with hATTR.

Objectives

To assess the neurological efficacy and safety of RNA therapeutics in hATTR patients.

Design

Systematic review and meta-analysis.

Data sources and methods

A systematic literature search was conducted on PubMed, Cochrane, and ClinicalTrials.gov from inception to August 14, 2024. Outcomes included changes from baseline in the Norfolk Quality of

SUBMITTER: Sajjad M 

PROVIDER: S-EPMC12612535 | biostudies-literature | 2025 Jan-Dec

REPOSITORIES: biostudies-literature