Histological effects of givinostat in boys with Duchenne muscular dystrophy
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- 作者：Paolo Betticaa ; 1 ; p.bettica@italfarmaco.com" class="auth_mail" title="E-mail the corresponding author ; Stefania Petrinib ; 1 ; Valentina D'Oriab ; Adele D'Amicoc ; Michela Catterucciac ; Marika Paned ; Serena Sivod ; Francesca Magrie ; Simona Brajkovice ; Sonia Messinaf ; g ; Gian Luca Vitag ; Barbara Gattia ; Maurizio Moggioh ; Pier Lorenzo Purii ; j ; Maurizio Rocchettik ; Giuseppe De Nicolaol ; Giuseppe Vitaf ; g ; Giacomo P. Comie ; Enrico Bertinic ; Eugenio Mercurid
- 关键词：Duchenne muscular dystrophy ; Givinostat ; Histone deacetylase inhibitor ; Histology ; Clinical trial
- 刊名：Neuromuscular Disorders
- 出版年：2016
- 出版时间：October 2016
- 年：2016
- 卷：26
- 期：10
- 页码：643-649
- 全文大小：952 K
文摘
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HDAC hyperactivity is part of DMD pathogenesis. Givinostat is an HDAC inhibitor.
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Givinostat increased muscle and decreased fibrosis and fat replacement in DMD boys.
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Givinostat was tolerated with platelet count reductions being the dose limiting AE.
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Givinostat counteracts the pathogenic events downstream of DMD genetic defect.