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Roth Capital Affirms Bioblast Pharma (ORPN) at 'Buy'; Extension Study Data Shows Sustainable Improvements

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BioBlast Pharma (ORPN) Announces Strong Results from HOPEMD Extension Study

September 12, 2016 6:10 AM EDT

Bioblast Pharma Ltd. (Nasdaq: ORPN) announced results of the Extension study for patients who completed HOPEMD, the Companys initial clinical trial for patients with Oculopharyngeal Muscular Dystrophy (OPMD). This Extension study was a multi-center, randomized, open-label study with a non-treatment concurrent control arm that enrolled 22 patients, 16 of whom continued to receive trehalose 90mg/mL IV solution while 6 were in the non-treatment group.

OPMD is a rare progressive muscle-wasting disease characterized by severe swallowing difficulties (dysphagia) leading to malnutrition, aspiration of food into the lungs and recurrent episodes of aspiration pneumonia, as... More