Histological effects of givinostat in boys with Duchenne muscular dystrophy

Highlights • HDAC hyperactivity is part of DMD pathogenesis. Givinostat is an HDAC inhibitor. • Givinostat increased muscle and decreased fibrosis and fat replacement in DMD boys. • Givinostat was tolerated with platelet count reductions being the dose limiting AE. • Givinostat counteracts the patho...

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Veröffentlicht in:	Neuromuscular disorders : NMD 2016-10, Vol.26 (10), p.643-649
Hauptverfasser:	Bettica, Paolo, Petrini, Stefania, D'Oria, Valentina, D'Amico, Adele, Catteruccia, Michela, Pane, Marika, Sivo, Serena, Magri, Francesca, Brajkovic, Simona, Messina, Sonia, Vita, Gian Luca, Gatti, Barbara, Moggio, Maurizio, Puri, Pier Lorenzo, Rocchetti, Maurizio, De Nicolao, Giuseppe, Vita, Giuseppe, Comi, Giacomo P, Bertini, Enrico, Mercuri, Eugenio
Format:	Artikel
Sprache:	eng
Schlagworte:	Adrenal Cortex Hormones - therapeutic use Carbamates - adverse effects Carbamates - therapeutic use Child Clinical trial Dose-Response Relationship, Drug Duchenne muscular dystrophy Givinostat Histology Histone deacetylase inhibitor Histone Deacetylase Inhibitors - adverse effects Histone Deacetylase Inhibitors - therapeutic use Humans Male Motor Activity - drug effects Muscle, Skeletal - drug effects Muscle, Skeletal - pathology Muscular Dystrophy, Duchenne - blood Muscular Dystrophy, Duchenne - drug therapy Muscular Dystrophy, Duchenne - pathology Neurology Platelet Count Treatment Outcome
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Zusammenfassung:	Highlights • HDAC hyperactivity is part of DMD pathogenesis. Givinostat is an HDAC inhibitor. • Givinostat increased muscle and decreased fibrosis and fat replacement in DMD boys. • Givinostat was tolerated with platelet count reductions being the dose limiting AE. • Givinostat counteracts the pathogenic events downstream of DMD genetic defect.
ISSN:	0960-8966 1873-2364
DOI:	10.1016/j.nmd.2016.07.002