Clinical use of immunosuppressants in Duchenne muscular dystrophy.
Duchenne muscular dystrophy (DMD) is a degenerative disease primarily affecting voluntary muscles with secondary consequences on heart and breathing muscles. DMD is an X-linked recessive disease that results in the loss of dystrophin, a key muscle protein. Inflammation can play different roles in DMD; it can be a secondary response to muscle degeneration, a primary cause of degeneration, or can contribute to the disease progression. Several immunosuppressants have been used with the aim to reduce the inflammation associated with DMD. Most recently, myoblast transplantation has shown the possibility to restore the dystrophin lack in the DMD patient's muscle fibers and this evidence has emphasized the importance of the use of immunosuppressants and the necessity of studying them and their secondary effects. The aim of this review is to analyze the main immunosuppressants drugs starting from the mdx mice experiments and concluding with the most recent human clinical studies.
Iannitti
Tommaso
T
Department of Biological and Biomedical Sciences, School of Life Sciences, Glasgow Caledonian University, Glasgow, UK. tommaso.iannitti@gmail.com
Capone
Stefania
S
Feder
David
D
Palmieri
Beniamino
B
D000818:Animals; D004195:Disease Models, Animal; D018450:Disease Progression; D006801:Humans; D007166:Immunosuppressive Agents / Q000009:adverse effects / Q000627:therapeutic use*; D007249:Inflammation / Q000188:drug therapy / Q000276:immunology / Q000473:pathology; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy* / Q000276:immunology / Q000473:pathology*; D016896:Treatment Outcome
D007166:Immunosuppressive Agents