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News Breaks
July 31, 2014
06:07 EDTORPNBio Blast Pharma 3.2M share IPO priced at $11.00
The deal range was $11.00-$13.00. Oppenheimer and Roth Capital acted as joint book running managers for the offering.
News For ORPN From The Last 14 Days
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June 29, 2015
08:20 EDTORPNBioBlast Pharma receives Orphan Drug Designation from EC for Cabaletta
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June 25, 2015
08:10 EDTORPNBio Blast Pharma receives ODD from EC for Cabaletta for the treatment of OPMD
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June 22, 2015
07:27 EDTORPNBio Blast announces positive preclinical proof-of-concept results of BBrm02
BioBlast Pharma announced positive preclinical in vitro and in vivo proof-of-concept study results for its Read-through drug candidate BBrm02 for Spinal Muscular Atrophy, or SMA. BBrm02 is a proprietary, intrathecal formulation of azithromycin. In a series of cell and mouse studies, BBrm02 was effective in creating high levels of full length SMN2 protein in the central nervous system resulting in a high degree of cellular and disease phenotypic rescues. Significant levels of full length SMN2 protein were detected following treatment with BBrm02 in several different patient cell lines representing different stages of disease severity. This SMN2 protein was tested for biological activity and was found to be highly functional. In two different mouse models of Spinal Muscular Atrophy, BBrm02 was effective in preventing weight loss, increasing animal survival, and in normalizing a range of neurological functions, including, among others, the ability of the mouse to return to four feet from a supine position and to walk on a narrow tube, both of which measure equilibrium and muscle strength.

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