Bioblast Pharma Announces Collaboration with Team Sanfilippo to Evaluate Trehalose for the Treatment of Sanfilippo Syndrome
BIO BLAST PHARMA LTD ORDINARY (ORPN)
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Source: GlobeNewswire
Tel Aviv, Israel, Feb. 14, 2019 (GLOBE NEWSWIRE) -- Bioblast Pharma Ltd. (NasdaqCM: ORPN) (“Bioblast”), a clinical-stage, orphan disease-focused biotechnology company, and Team Sanfilippo (“TSF”), a nonprofit medical research foundation founded by parents of children with Sanfilippo Syndrome today announced a clinical collaboration to evaluate the safety and efficacy of Bioblast’s proprietary Trehalose solution for the treatment of patients with Sanfilippo syndrome. Under the terms of the collaboration, TSF plans to conduct an open label study in up to 20 Sanfilippo patients, which will be followed for safety and efficacy. If successful, based on the results of such study, Bioblast may determine to continue the development of Trehalose towards a pivotal study for this extra rare disease while maintaining full commercial rights. TSF received FDA approval for this study was last week. "We are excited to be working with TSF to explore the potential of Trehalose in this ra
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