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One-'triq' pony?

Exelixis Inc. will downsize by 70 percent in the wake of the pivotal phase III failure by Cometriq (cabozantinib) in prostate cancer (PC), and awaits results that are due next year from the late-stage experiment in renal cell carcinoma. more »

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Promising compounds 'profoundly alter' disease course in SMA

Scientists from Roche Pharma Research and Early Development (pRED) and PTC Therapeutics Inc., with colleagues in academia, reported on small-molecule drugs that improved the symptoms of spinal muscular atrophy (SMA) by altering the splicing of the protein SMN2.

In mouse models of SMA, the compounds "profoundly altered the course of the disease," PTC Therapeutics CEO and co-author Stuart Peltz told BioWorld Today.



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