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Globavir Biosciences Inc. formally threw its hat into the Ebola ring, making known the firm's plan to develop the preclinical candidate GBV006 as a potential remedy for the virulent outbreak in West Africa. 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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