Prepare for the era of gene therapy for Duchenne muscular dystrophy

Abstract

In recent years, the clinical trials of Duchenne muscular dystrophy (DMD) gene therapy in foreign countries have proved to be of certain curative effect and good safety. In China, DMD, with a large number of patients, has been introduced into the first rare diseases catalog, indicating that gene therapy will soon enter our country. It is a revolutionary treatment for DMD, and thus clinicians, patients and their families, relevant government departments, charity foundations and media shall prepare for the era of DMD gene therapy, which will benefit for DMD patients.

DOI: 10.3969/j.issn.1672-6731.2019.05.002