New Therapeutic Approaches to Spinal Muscular Atrophy
Author:
Publisher
Springer Science and Business Media LLC
Subject
Neurology (clinical),General Neuroscience
Link
http://link.springer.com/content/pdf/10.1007/s11910-011-0240-9.pdf
Reference65 articles.
1. Zerres K, Wirth B, Rudnik-Schoneborn S. Spinal muscular atrophy-clinical and genetic correlations. Neuromuscular Disorders. 1997;7:202–7.
2. • Pruss RM, Giraudon-Paoli M, Morozova S, et al. Drug discovery and development for spinal muscular atrophy: lessons from screening approaches and future challenges for clinical development. Future Med Chem. 2010;2:1429–40. In this excellent and comprehensive review, Pruss et al. review lessons learned and discuss potential pitfalls using screening approaches to identify pharmacologic therapies for SMA using cell-based assays, including examples from their experience with both SMN- and non-SMN-directed strategies.
3. • Shababi M, Mattis VB, Lorson CL. Therapeutics that directly increase SMN expression to treat spinal muscular atrophy. Drug News Perspect. 2010;23:475–82. This review nicely summarizes therapeutic approaches including nucleic acid-based and pharmacologic therapeutics that hold promise as potential treatment options in SMA.
4. Kolb SJ, Kissel JT. Spinal muscular atrophy: a timely review. Arch Neurol. 2011;68:979–84.
5. Lunn JS, Sakowski SA, Federici T, et al. Stem cell technology for the study and treatment of motor neuron diseases. Regen Med. 2011;6:201–13.
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