Targeted Therapies for Hereditary Peripheral Neuropathies: Systematic Review and Steps Towards a 'treatabolome'.

ABSTRACT:

Background

Hereditary peripheral neuropathies are inherited disorders affecting the peripheral nervous system, including Charcot-Marie-Tooth disease, familial amyloid polyneuropathy and hereditary sensory and motor neuropathies. While the molecular basis of hereditary peripheral neuropathies has been extensively researched, interventional trials of pharmacological therapies are lacking.

Objective

We collated evidence for the effectiveness of pharmacological and gene-based treatments for hereditary peripheral neuropathies.

Methods

We searched several databases for randomised controlled trials (RCT), observational studies and case reports of therapies in hereditary peripheral neuropathies. Two investigators extracted and analysed the data independently, assessing study

SUBMITTER: Jennings MJ 

PROVIDER: S-EPMC8203235 | biostudies-literature | 2021

REPOSITORIES: biostudies-literature