givinostat (Duvyzat)
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Indications
- treatment of Duchenne Muscular Dystrophy (DMD) in boys >= 6 years of age
Dosage
Mechanism of action
- histone deacetylase (HDAC) inhibitor
- prevents gene translation through changing the 3D folding of cell DNA
- inhibiting HDACs promotes muscle repair, reduces inflammation, & decreases both muscle fibrosis & fat accumulation in patients
More general terms
References
- ↑ FDA NEWS RELEASE. March 21, 2024 FDA Approves Nonsteroidal Treatment for Duchenne Muscular Dystrophy https://www.fda.gov/news-events/press-announcements/fda-approves-nonsteroidal-treatment-duchenne-muscular-dystrophy
- ↑ Shaw ML Givinostat Receives Full FDA Approval for Duchenne Muscular Dystrophy American Journal of Managed Care (AJMC). 2024. March 21. https://www.ajmc.com/view/givinostat-receives-full-fda-approval-for-duchenne-muscular-dystrophy
- ↑ Mercuri E, Vilchez JJ, Boespflug-Tanguy O et al Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Neurol. 2024 Apr;23(4):393-403. PMID: https://www.ncbi.nlm.nih.gov/pubmed/38508835 Clinical Trial. https://www.thelancet.com/journals/laneur/article/PIIS1474-4422(24)00036-X/fulltext