Safety and Efficacy of Givinostat for Patients with Muscular Dystrophy: A Systematic Review.

ABSTRACT:

Introduction

Muscular dystrophy (MD) refers to a group of genetic disorders leading to progressive weakness and degeneration of skeletal muscle. Among them, Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) are the most common types. Histone deacetylase inhibitors (i.e., givinostat) is a recently approved therapy for DMD. In this systematic review, we aimed to evaluate givinostat in MD patients regarding safety and efficacy.

Methods

A systematic review was performed according to the PRISMA guidelines by searching through Medline, Embase, Cochrane Library, and Web of Science. Only randomized control trials comparing givinostat vs. placebo or other therapies are included. The primary outcomes were motor function alteration and histopathologic muscle changes

SUBMITTER: Das P 

PROVIDER: S-EPMC12503821 | biostudies-literature | 2025 Aug

REPOSITORIES: biostudies-literature