Fabry Disease: Current and Novel Therapeutic Strategies. A Narrative Review.

ABSTRACT:

Background

Fabry disease (FD) is an inherited lysosomal storage disorder, leading to multisystemic manifestations and causing significant morbidity and mortality.

Objective

The aim of this narrative review is to present the current and novel therapeutic strategies in FD, including symptomatic and specific treatment options.

Methods

A systematic literature search was conducted to identify relevant studies, including completed and ongoing randomized-controlled clinical trials (RCTs), prospective or retrospective cohort studies, case series and case reports that provided clinical data regarding FD treatment.

Results

A multidisciplinary symptomatic treatment is recommended for FD patients, personalized according to disease manifestations and their severity. During

SUBMITTER: Palaiodimou L 

PROVIDER: S-EPMC10207921 | biostudies-literature | 2023

REPOSITORIES: biostudies-literature