Gunnar M Buyse
Affiliation: Katholieke Universiteit Leuven
- Idebenone treatment in Friedreich's ataxia: neurological, cardiac, and biochemical monitoringG Buyse
Department of Pediatric Neurology, University Hospitals Leuven, Belgium
Neurology 60:1679-81. 2003..Idebenone reduced erythrocyte protoporphyrin IX levels in five of six patients with elevated baseline levels; however, changes did not consistently relate to cardiac improvement...
- CINRG pilot trial of oxatomide in steroid-naïve Duchenne muscular dystrophyGunnar M Buyse
University Hospitals KU Leuven, Leuven, Herestraat 49, B 3000 Leuven, Belgium
Eur J Paediatr Neurol 11:337-40. 2007..A modest mitigation of strength deterioration over time cannot be excluded...
- Effects of glucocorticoids and idebenone on respiratory function in patients with duchenne muscular dystrophyGunnar M Buyse
Department of Child Neurology, University Hospitals Leuven, Belgium
Pediatr Pulmonol 48:912-20. 2013..The impact of standard care glucocorticoids on respiratory function will have to be considered in the planning of future interventional trials in DMD...
- Idebenone as a novel, therapeutic approach for Duchenne muscular dystrophy: results from a 12 month, double-blind, randomized placebo-controlled trialGunnar M Buyse
Child Neurology, University Hospitals Leuven, Leuven, Belgium
Neuromuscul Disord 21:396-405. 2011..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...
- Regulated granule trafficking in platelets and neurons: a common molecular machineryChristophe Goubau
Center for Molecular and Vascular Biology, University of Leuven, Leuven, Belgium
Eur J Paediatr Neurol 17:117-25. 2013..This review provides evidence that platelet research can be expanded from traditional studies of isolated thrombopathies to the field of neuropathologies that include a platelet secretion defect...
- The contribution of platelet studies to the understanding of disease mechanisms in complex and monogenetic neurological disordersChristophe Goubau
Center for Molecular and Vascular Biology, University of Leuven, Leuven, Belgium Department of Child Neurology, University Hospitals Leuven, Leuven, Belgium
Dev Med Child Neurol 56:724-31. 2014....
- Efficacy of idebenone on respiratory function in patients with Duchenne muscular dystrophy not using glucocorticoids (DELOS): a double-blind randomised placebo-controlled phase 3 trialGunnar M Buyse
University Hospitals Leuven, Leuven, Belgium Electronic address
Lancet 385:1748-57. 2015..Based on preclinical and phase 2 evidence, we assessed the efficacy and safety of idebenone in young patients with Duchenne muscular dystrophy who were not taking concomitant glucocorticoids...
- Long-term blinded placebo-controlled study of SNT-MC17/idebenone in the dystrophin deficient mdx mouse: cardiac protection and improved exercise performanceGunnar M Buyse
Department of Pediatric Neurology, University Hospitals Leuven, Herestraat, Leuven, Belgium
Eur Heart J 30:116-24. 2009..We have performed such a trial of SNT-MC17/idebenone in the mdx mouse model of DMD, based on the drug's potential to improve mitochondrial respiratory chain function and reduce oxidative stress...
- Homozygosity for aquaporin 7 G264V in three unrelated children with hyperglyceroluria and a mild platelet secretion defectChristophe Goubau
Department of Cardiovascular Sciences, Center for Molecular and Vascular Biology, University of Leuven, Leuven, Belgium
Genet Med 15:55-63. 2013..AQP7-deficient mice develop obesity, insulin resistance, and hyperglyceroluria. However, AQP7's pathophysiologic role in humans is not yet known...