Affiliation: Children's National Medical Center
- Translating mighty mice into neuromuscular therapeutics: is bigger muscle better?Eric P Hoffman
Research Center for Genetic Medicine, Children s National Medical Center, 111 Michigan Ave NW, Washington, DC 20010, USA
Am J Pathol 168:1775-8. 2006
- Collaborative translational research leading to multicenter clinical trials in Duchenne muscular dystrophy: the Cooperative International Neuromuscular Research Group (CINRG)Diana M Escolar
Research Center for Genetic Medicine CRI III, Children s National Medical Center, George Washington University, 111 Michigan Avenue, NW, Washington, DC 20010, USA
Neuromuscul Disord 12:S147-154. 2002....
- CINRG randomized controlled trial of creatine and glutamine in Duchenne muscular dystrophyDiana M Escolar
Children s National Medical Center, George Washington University, Washington, DC, USA
Ann Neurol 58:151-5. 2005..Creatine and glutamine were well tolerated...
- Reliable surrogate outcome measures in multicenter clinical trials of Duchenne muscular dystrophyJill E Mayhew
Research Center for Genetic Medicine, Children s National Medical Center, 111 Michigan Avenue NW, Washington, DC 20010, USA
Muscle Nerve 35:36-42. 2007..61). These data suggest that primary surrogate outcome measures in large multicenter clinical trials in DMD should use QMT, FVC, or time function tests to obtain maximum power and greatest sensitivity...
- Functional and molecular effects of arginine butyrate and prednisone on muscle and heart in the mdx mouse model of Duchenne Muscular DystrophyAlfredo D Guerron
Center for Genetic Medicine Research, Children s National Medical Center, Department of Integrative Systems Biology, George Washington University School of Medicine and Health Sciences, Washington, DC, USA
PLoS ONE 5:e11220. 2010..One of the proposed strategies is to use drugs that are known to act by multiple different mechanisms including inducing of homologous fetal form of adult genes, for example utrophin in place of dystrophin...
- Challenges in drug development for muscle disease: a stakeholders' meetingJerry R Mendell
Columbus Children s Research Institute, and Ohio State University, 700 Children s Drive, Columbus, OH 43235, USA
Muscle Nerve 35:8-16. 2007..The meeting provided a format for communication for diverse disciplines that usually have no common meeting ground, helping to lay the foundation for bringing products to market in a timely fashion...
- 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...