enzyme replacement therapy

  • Agalsidase alfa versus agalsidase beta for the treatment of Fabry disease: an international cohort study
    Maarten Arends, Marieke Biegstraaten, Christoph Wanner, Sandra Sirrs, Atul Mehta, Perry M Elliott, Daniel Oder, Oliver T Watkinson, Daniel G Bichet, Aneal Khan, Mark Iwanochko, Frédéric M Vaz, André B P van Kuilenburg, Michael L West, Derralynn A Hughes, Carla E M Hollak
  • Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study
    Derralynn A Hughes, Kathleen Nicholls, Suma P Shankar, Gere Sunder-Plassmann, David Koeller, Khan Nedd, Gerard Vockley, Takashi Hamazaki, Robin Lachmann, Toya Ohashi, Iacopo Olivotto, Norio Sakai, Patrick Deegan, David Dimmock, François Eyskens, Dominique P Germain, Ozlem Goker-Alpan, Eric Hachulla, Ana Jovanovic, Charles M Lourenco, Ichiei Narita, Mark Thomas, William R Wilcox, Daniel G Bichet, Raphael Schiffmann, Elizabeth Ludington, Christopher Viereck, John Kirk, Julie Yu, Franklin Johnson, Pol Boudes, Elfrida R Benjamin, David J Lockhart, Carrolee Barlow, Nina Skuban, Jeffrey P Castelli, Jay Barth, Ulla Feldt-Rasmussen