Tag Archives: Niemann-Pick disease

January, 2016

June, 2015

  • 8 June

    Breakthrough Status Granted to Genzyme’s Rare Enzyme Replacement Drug

    Genzyme, a Sanofi company, recently announced that its investigational enzyme-replacement therapy was granted Breakthrough Therapy designation from US health regulators. The company said that the agency granted the status to its olipudase alfa for the treatment of patients with nonneurological manifestations of acid sphingomyelinase deficiency (ASMD), also known as Niemann-Pick …