A number of drugs for spinal muscular atrophy (SMA) is being developed right now. Among the most promising ones seems to be the ISIS Pharmaceuticals SMN-Rx an antisense drug designed to potentially treat all types of childhood SMA by altering the SMN2 gene, that leads to the increased production of fully functional SMN protein. ISIS SMN-Rx entered phase 1 clinical trials last year and is set to finish phase 1 by the end of this year.
October 2012 issue of Journal Cell Biology brings an insightful article on how the antisense drug was developed and what it does. The whole article can be found at this link.
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