TEL AVIV, Israel, Jan. 8, 2015 (GLOBE NEWSWIRE) — BioBlast Pharma Ltd. (ORPN), a clinical-stage biotechnology company committed to developing meaningful therapies for patients with rare and ultra-rare genetic diseases, announced today preliminary findings from its HOPEMD Phase 2/3 study investigating Cabaletta (trehalose) in patients with Oculopharyngeal Muscular Dystrophy (OPMD).

The multicenter study is testing the safety and tolerability of Cabaletta in patients suffering from OPMD – a progressive, debilitating genetic disease – across three centers in Israel, Canada and the U.S.