Bio Blast Pharma Ltd. (ORPN) announced it has been granted Orphan Drug Designation by the U.S. FDA for Cabaletta for the treatment of Spinocerebellar Ataxia Type 3 (known as SCA3 and Machado Joseph disease). This is the second indication for which Bio Blast's Cabaletta has received such designation.
Cabaletta is a chemical chaperone that protects against pathological processes in cells. The company noted that Cabaletta has demonstrated efficacy in preclinical cells and animal models of SCA3 and other PolyA/PolyQ diseases, including Occulopharyngeal Muscular Dystrophy and Spino bulbar cerebellar ataxia. Bio Blast plans to make clinical progress in each of these indications in 2015.
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June 05, 2026 16:18 ET A busy week for economic news flow saw a slew of reports being released that reflected the trends in the U.S. labor market. In Europe, economic growth and inflation data gained attention as the European Central Bank and Bank of England head for policy session later in the month. In Asia, the monetary policy session of the Indian central bank was in focus as the country, a major oil importer, reels under the pressures of a weaker rupee and rising inflation.