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Next steps: Meeting with FDA, EMA

Amicus Therapeutics Inc.'s shares rocketed 20.4 percent Wednesday as the company delivered positive top-line data from a second phase III study of migalastat, its oral small-molecule chaperone for Fabry disease patients. 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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