Five Strategies for Treating Neuromuscular Disease
Quest (MDA) | July 2011 | By Amy Labbe
- This article describes five leading therapeutic strategies currently being tried in neuromuscular disease research: antisense oligonucleotides, stem cells, small molecules, protein therapy and gene therapy.
- These strategies are relevant in ALS, Becker MD, centronuclear myopathies/myotubular myopathy, Charcot-Marie-Tooth disease, congenital MD, Dejerine-Sottas disease, distal MD (Miyoshi myopathy), Duchenne MD, facioscapulohumeral MD, Friedreich’s ataxia, limb-girdle MD, myotonic MD and spinal muscular atrophy.
- The strategies were the focus of an MDA scientific conference in March 2011 that was designed to help advance these strategies from “microscope to marketplace.”
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