دورية أكاديمية
Givinostat in DMD: Results of the EPIDYS Study with Particular Attention to NSAA.
| العنوان: | Givinostat in DMD: Results of the EPIDYS Study with Particular Attention to NSAA. |
|---|---|
| المؤلفون: | Mercuri, E., Brogna, C., Mah, J. K., Goemans, N., Niks, E., Müller-Felber, W., Blaschek, A., Schmidt, U. Schara, Johannsen, J., Cazzaniga, S., Bettica, P., McDonald, C. |
| المصدر: | Neuropediatrics; 2023 Supplement 1, Vol. 54, pS1-S32, 32p |
| مصطلحات موضوعية: | PHYSICAL mobility, DUCHENNE muscular dystrophy, HISTONE deacetylase inhibitors, MUSCLE strength |
| مستخلص: | The article discusses the results of the EPIDYS study, which evaluated the efficacy and safety of Givinostat, a novel histone deacetylase inhibitor, in ambulant patients with Duchenne muscular dystrophy (DMD). The study found that Givinostat demonstrated a statistically significant difference in the time to climb 4 standard stairs compared to the placebo. The treatment also showed positive effects on secondary endpoints, including physical function, muscle strength, and muscle fat fraction. Givinostat was generally well-tolerated, with reversible side effects related to thrombocytopenia and hypertriglyceridemia. The study concludes that Givinostat has clinically important effects on physical function in boys with DMD. [Extracted from the article] |
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| قاعدة البيانات: | Complementary Index |
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