Research Progress on the Efficacy and Safety of Deflazacort in the Treatment of Duchenne Muscular Dystrophy
- VernacularTitle:地夫可特治疗杜氏肌营养不良有效性及安全性研究进展
- Author:
Tingting XU
1
;
Wei ZUO
1
;
Xin LIU
1
;
Shaohong WANG
1
;
Zhuo SUN
1
;
Junmei SHANG
1
;
Luyao QIAO
1
;
Bo ZHANG
1
Author Information
- Publication Type:Journal Article
- Keywords: Duchenne muscular dystrophy; deflazacort; clinical efficacy; adverse reactions
- From: JOURNAL OF RARE DISEASES 2025;4(2):248-257
- CountryChina
- Language:Chinese
- Abstract: Deflazacort,as a glucocorticoid medication,is conductive to improving motor function and muscle strength,delaying the loss of ambulation,enhancing pulmonary function,reducing the risk of scoliosis,slowing the progression of cardiomyopathy,and increasing survival rates in patients with Duchenne muscular dystrophy(DMD).In February 2017,the U.S.Food and Drug Administration(FDA)approved deflazacort for the treatment of DMD.In May 2024,deflazacort entered Peking Union Medical College Hospital for desig-nated use through the ＂ temporary import＂ pathway.This article provides an overview of deflazacort from the perspectives of its mechanism of action,pharmacokinetics,clinical efficacy,and adverse effects,aiming to offer a reference for its rational and safe application in clinical practice.
