Efficacy and the Safety of Granulocyte Colony-Stimulating Factor Treatment in Patients with Muscular Dystrophy: A Non-Randomized Clinical Trial.

ABSTRACT:

Introduction

The current standard treatment for patients with Duchenne muscular dystrophy (DMD) involves corticosteroids. Granulocyte colony-stimulating factor (G-CSF) induces the proliferation of satellite cells and myoblasts and, in turn, muscle regeneration. Beneficial effects of G-CSF were also described for skeletal muscle disorders.

Aim

We assessed the safety and effects of using G-CSF to promote muscle strength in patients with DMD.

Materials and methods

Inclusion criteria were as follows: patients aged 5-15 years with diagnosed with DMD confirmed by genetic test or biopsy. Fourteen patients were treated with steroids, and their use was not changed in this study. Diagnoses were confirmed by genetic tests: deletions were detected in 11 patients and duplications

SUBMITTER: Sienkiewicz D 

PROVIDER: S-EPMC5662550 | biostudies-literature | 2017

REPOSITORIES: biostudies-literature