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'Quicker expansion of our diabetic nephropathy portfolio'
LONDON – The last five years have seen a dramatic increase in the number of discovery collaborations established by pharma companies with academic researchers and biotechs. 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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