Muscular Dystrophy is estimated to affect 150,000 patients in the USA, Europe and Japan and is passed from parent to child with each subsequent generation experiencing more severe disease earlier in life.
Isis will be responsible for the discovery of a lead antisense drug candidate targeting DMPK for the treatment of DM1 (muscular dystrophy in adults)
Steven Holtzman, head of corporate development at Biogen said that “the unmet need is great and there are currently no therapies to slow or stop progression of the disease” He also said DM1 has “an identifiable genetic cause, the program fits with our mission to bring innovative therapies to patients with serious neurologic diseases, and Isis’ antisense compound has the potential to make a real difference”
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