Collaborative translational research leading to multicenter clinical trials in Duchenne muscular dystrophy: The Cooperative International Neuromuscular Research Group (CINRG)

Diana M. Escolar, Erik K Henricson, Livia Pasquali, Ksenija Gorni, Eric P. Hoffman

Research output: Contribution to journalArticle

13 Scopus citations

Abstract

Progress in the development of rationally based therapies for Duchenne muscular dystrophy has been accelerated by encouraging multidisciplinary, multi-institutional collaboration between basic science and clinical investigators in the Cooperative International Research Group. We combined existing research efforts in pathophysiology by a gene expression profiling laboratory with the efforts of animal facilities capable of conducting high-throughput drug screening and toxicity testing to identify safe and effective drug compounds that target different parts of the pathophysiologic cascade in a genome-wide drug discovery approach. Simultaneously, we developed a clinical trial coordinating center and an international network of collaborating physicians and clinics where those drugs could be tested in large-scale clinical trials. We hope that by bringing together investigators at these facilities and providing the infrastructure to support their research, we can rapidly move new bench discoveries through animal model screening and into therapeutic testing in humans in a safe, timely and cost-effective setting.

Original languageEnglish (US)
JournalNeuromuscular Disorders
Volume12
Issue numberSUPPL.
DOIs
StatePublished - Oct 2002
Externally publishedYes

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Keywords

  • Cooperative group
  • Cooperative International Neuromuscular Research Group (CINRG)
  • Duchenne muscular dystrophy
  • Gene expression profiling
  • High throughput screening
  • Multicenter clinical trial
  • Phamacologic screening
  • Toxicity testing

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Neurology
  • Clinical Neurology
  • Genetics(clinical)

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