Duchenne muscular dystrophy: Current state and therapeutic perspectives

被引：0

作者：

Le Guen, Y. T. ^{[1
]}

Le Gall, T. ^{[1
]}

Laurent, V ^{[1
]}

D'Arbonneau, F. ^{[1
]}

Braun, S. ^{[2
]}

Montier, T. ^{[1
,3
]}

机构：

[1] Univ Bretagne Occidentale, INSERM, Genet Genom Fonct & Biotechnol, EFS,UMR 1078, 22 Rue Camille Desmoulins,CS 93837, F-29238 Brest 3, France

[2] AFM Telethon, 1 Rue Int,BP59, F-91002 Evry, France

[3] CHRU Brest, Serv Genet Med & Biol Reprod, Ctr Reference Malad Rares Malad Neuromusculaires, F-29200 Brest, France

来源：

BULLETIN DE L ACADEMIE NATIONALE DE MEDECINE | 2021年 / 205卷 / 05期

关键词：

Muscular Dystrophy; Duchenne; Therapeutics; Gene Therapy; GENE-THERAPY; FEMALE CARRIERS; MUSCLE; GLUCOCORTICOIDS; CHILDREN; VECTORS; IMPACT;

D O I：

10.1016/j.banm.2020.10.019

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

Duchenne muscular dystrophy is one of the most frequent muscular dystrophies, with 250,000 affected people worldwide. It results from mutations occurring in the dystrophin gene, the largest known in the human genome. Despite current therapies allow for a better clinical prognosis, the life span and the quality of life of patients still remain clearly lower than those in the general population. New therapeutic approaches are currently investigated, giving new perspectives and hopes for the care of this severe disease. (C) 2021 l'Academie nationale de medecine. Published by Elsevier Masson SAS. All rights reserved.

引用

页码：509 / 518

页数：10

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