Safety and effectiveness of givinostat in boys with Duchenne muscular dystrophy: a plain language summary of the EPIDYS clinical trial
Le résumé fourni par la source
What is this summary about? This is a plain language summary describing the EPIDYS study results. This study evaluated how safe and effective givinostat was for boys with Duchenne muscular dystrophy (DMD). What happened in the EPIDYS study? Between June 6, 2017, and February 22, 2022, a total of 179 boys entered EPIDYS: 118 were randomly assigned to receive givinostat, and 61 received placebo (a liquid that looks the same but has no medicine in it). Researchers evaluated the efficacy of givinostat, which is how well the medicine worked, by measuring the boys’ muscle strength and function before and during treatment (givinostat or placebo). Results from 120 boys who met the target fat and muscle range were used to assess both the safety and efficacy of givinostat; the remaining 59 boys outside the target range were assessed for safety only. What were the results? Boys with DMD who took givinostat for 72 weeks had less decline in their muscle strength and function (measured by the 4-stair climb test) than those who took placebo. Most of the negative health events that occurred during treatment were mild to moderate in severity and happened about equally in both groups. The most common treatment-related negative reactions were low platelet count (blood clotting cells), high triglyceride (blood fat) levels, and diarrhea, which were more frequent with givinostat treatment. What do the results of the study mean? The EPIDYS study showed that givinostat might help boys with DMD by slowing loss of muscle function as measured by the 4-stair climb test, and no major safety concerns were observed. How to say (download PDF and double click sound icon to play sound)… Duchenne muscular dystrophy: Doo-SHEN MUH-skyuh-lr DIS-truh-fee Dystrophin: di-STRUH-fin Givinostat: giv-IN-oh-stat/jee-IN-oh-stat Histone deacetylase: HIS-tone dee-uh-SEH-tih-lays Deflazacort: dee-FLAYZ-uh-kort Corticosteroid: kor-tih-koh-STAIR-oyd This is an abstract of the Plain Language Summary of Publication article. View the full Plain Language Summary PDF of this article to read the full-text Link to original article here
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