Tag Archives: enzyme replacement therapy

October, 2018

April, 2018

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 …

April, 2015

  • 8 April

    FDA Accepts ArmaGen’s IND for its Hurler Syndrome Candidate

    Privately-held biotech ArmaGen, Inc. announced today that US health regulators have accepted for review its investigational New Drug (IND) application for its drug for a rare lysosomal storage disease. The company, focused on developing novel therapies to treat severe neurological disorders, said that the US Food and Drug Administration (FDA) …