Tag Archives: Fabry disease

August, 2018

September, 2017

  • 22 September

    Avrobio Expands Rare Disease Pipeline with Gene Therapy to Treat Pompe Disease

    CAMBRIDGE, Mass.–(BUSINESS WIRE)–AVROBIO, Inc., a clinical-stage biotechnology company developing transformative, life-changing gene therapies for rare diseases, today announced the expansion of its pipeline to Pompe disease. This pre-clinical program becomes AVROBIO’s third gene therapy for Lysosomal Storage Disorders (LSDs), following on the heels of the Company’s Phase 1 Fabry program …

February, 2017

April, 2015

  • 29 April

    Genzyme’s Fabry Disease Drug Granted FDA Fast Track Status

    US health regulators have granted a speedy review for Genzyme’s investigational drug for treatment of a rare lysosomal storage disorder. Genzyme, a Sanofi company, announced that the US Food and Drug Administration (FDA) has granted Fast Track designation for the development of GZ/SAR402671, a new investigational oral substrate reduction therapy …

March, 2015