TY - JOUR
T1 - Collaborative translational research leading to multicenter clinical trials in Duchenne muscular dystrophy
T2 - The Cooperative International Neuromuscular Research Group (CINRG)
AU - Escolar, Diana M.
AU - Henricson, Erik K.
AU - Pasquali, Livia
AU - Gorni, Ksenija
AU - Hoffman, Eric P.
N1 - Funding Information:
The CINRG network is supported by grants RR13297 and RR16181-01 from the NIH National Center for Research Resources, Muscular Dystrophy Association, Association Francaise contre les Myopathies (AFM), Dutch Parent Project, The Serving Up A Cure Committee, The Foundation to Eradicate Duchenne, The Doug and Lynn Parsons Foundation and the Stichting-Porticus Foundation.
PY - 2002/10
Y1 - 2002/10
N2 - 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.
AB - 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.
KW - Cooperative International Neuromuscular Research Group (CINRG)
KW - Cooperative group
KW - Duchenne muscular dystrophy
KW - Gene expression profiling
KW - High throughput screening
KW - Multicenter clinical trial
KW - Phamacologic screening
KW - Toxicity testing
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U2 - 10.1016/S0960-8966(02)00094-9
DO - 10.1016/S0960-8966(02)00094-9
M3 - Article
C2 - 12206809
AN - SCOPUS:0036823509
SN - 0960-8966
VL - 12
SP - S147-S154
JO - Neuromuscular Disorders
JF - Neuromuscular Disorders
IS - SUPPL.
ER -