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Treatment extends survival in mouse model of spinal muscular atrophy

an ameliorate the disease in mice." While the results are exciting, there are still no studies that have proven the effectiveness of HDAC inhibitors in humans, she cautions.

The investigators are now testing whether treatment with TSA earlier in the disease process will work better than the delayed treatment in this study. They also plan to test other HDAC inhibitors in mice and to study exactly how the drugs influence the disease process. While TSA is expensive to produce and it is not approved for clinical use, similar drugs being developed to treat cancer and other diseases may be useful for treating SMA, Dr. Sumner says.


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Contact: Natalie Frazin
301-496-5924
NIH/National Institute of Neurological Disorders and Stroke
22-Feb-2007


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