Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The . gov means it’s official. Federal government websites often end in . gov or . mil VSports app下载. Before sharing sensitive information, make sure you’re on a federal government site. .

Https

The site is secure. The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely V体育官网. .

Clinical Trial
. 2011 Jun;21(6):396-405.
doi: 10.1016/j.nmd.2011.02.016. Epub 2011 Mar 23.

Idebenone as a novel, therapeutic approach for Duchenne muscular dystrophy: results from a 12 month, double-blind, randomized placebo-controlled trial

Gunnar M Buyse  1 , Nathalie Goemans, Marleen van den Hauwe, Daisy Thijs, Imelda J M de Groot, Ulrike Schara, Berten Ceulemans, Thomas Meier, Luc Mertens
Affiliations
Clinical Trial

Idebenone as a novel, therapeutic approach for Duchenne muscular dystrophy: results from a 12 month, double-blind, randomized placebo-controlled trial

Gunnar M Buyse et al. Neuromuscul Disord. 2011 Jun.

V体育官网入口 - Abstract

Early mortality in Duchenne muscular dystrophy (DMD) is related to cardiac and respiratory complications. A phase IIa double-blind randomized placebo-controlled clinical trial was conducted to investigate the tolerability and efficacy of idebenone therapy in children with DMD. Twenty-one DMD patients (aged 8-16 years) were randomly assigned to daily treatment with 450 mg idebenone (Catena®) (n=13) or placebo (n=8) for 12 months. All subjects completed the study and idebenone was safe and well tolerated. Idebenone treatment resulted in a trend (p=0. 067) to increase peak systolic radial strain in the left ventricular inferolateral wall, the region of the heart that is earliest and most severely affected in DMD. A significant respiratory treatment effect on peak expiratory flow was observed (p=0 VSports手机版. 039 for PEF and p=0. 042 for PEF percent predicted). Limitations of this study were the small sample size, and a skewed age distribution between treatment groups. Data from this study provided the basis for the planning of a confirmatory study. .

PubMed Disclaimer

Publication types

MeSH terms

LinkOut - more resources (V体育安卓版)