BioBlast Pharma (NASDAQ:ORPN) of Tel Aviv has been granted Orphan Drug status by the U.S. Food & Drug Administration (FDA) for its product candidate Cabaletta for the treatment of Spinocerebellar Ataxia Type 3 (also known as SCA3 or Machado-Joseph disease. This is the second ...

BioBlast Pharma Ltd, a clinical stage biotechnology company focusing on therapies for rare genetic diseases, is celebrating its recent IPO and the end of its quiet period with a visit to the NASDAQ MarketSite in Times Square. CEO Dr. Dalia ...