Document Detail

Drug discovery and development for spinal muscular atrophy: lessons from screening approaches and future challenges for clinical development.
MedLine Citation:
PMID:  21426138     Owner:  NLM     Status:  In-Data-Review    
Spinal muscular atrophy (SMA) is a progressive pediatric neuromuscular disease. Because disease severity is related to survival motor neuron (SMN) protein levels, increasing SMN production from the SMN2 gene has been a major SMA drug-discovery strategy. Cell-based assays using neuronal cell lines and cells from SMA patients have identified compounds that can increase SMN protein expression. Our experience of using such an assay signaled potential risks to be avoided through the use of appropriate secondary assays. In addition to the 'SMN2' approach, compensating for decreased SMN protein or neuroprotection are also potential SMA drug-discovery strategies. SMA clinical trials are now a reality; however, trial design in a slowly progressing rare disease such as SMA will present an interesting future challenge.
Rebecca M Pruss; Marc Giraudon-Paoli; Svetlana Morozova; Patrick Berna; Jean-Louis Abitbol; Thierry Bordet
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Publication Detail:
Type:  Journal Article    
Journal Detail:
Title:  Future medicinal chemistry     Volume:  2     ISSN:  1756-8927     ISO Abbreviation:  Future Med Chem     Publication Date:  2010 Sep 
Date Detail:
Created Date:  2011-03-23     Completed Date:  -     Revised Date:  -    
Medline Journal Info:
Nlm Unique ID:  101511162     Medline TA:  Future Med Chem     Country:  England    
Other Details:
Languages:  eng     Pagination:  1429-40     Citation Subset:  IM    
Trophos, Parc Scientifique de Luminy, Case 931, 13288 Marseille, France.
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