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Biotech beat goes on
The fact that U.S. markets were preparing to enter the long Labor Day weekend didn't stop two more biotechs from stepping out with IPOs. Rhythm Pharmaceuticals Inc. is seeking to raise $86.25 million. Meanwhile, Dermira Inc. set a goal of raising $75 million.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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