Long-term outcomes with agalsidase alfa enzyme replacement therapy: Analysis using deconstructed composite events

Por Beck, Michael, Hughes, Derralynn, Kampmann, Christoph, Pintos-Morell, Guillem, Ramaswami, Uma, West, Michael L., Giugliani, Roberto
Publicado no Mol Genet Metab Rep (2017)

Uncertain Diagnosis of Fabry Disease in Patients with Neuropathic Pain, Angiokeratoma or Cornea Verticillata: Consensus on the Approach to Diagnosis and Follow-Up

Por van der Tol, L., Cassiman, David, Houge, Gunnar, Janssen, Mirian C., Lachmann, Robin H, Linthorst, Gabor E, Ramaswami, Uma, Sommer, Claudia, Tøndel, Camilla, West, Michael L, Weidemann, Frank, Wijburg, Frits A, Svarstad, Einar, Hollak, Carla EM, Biegstraaten, Marieke
Publicado no JIMD Rep (2014)

Agalsidase alfa versus agalsidase beta for the treatment of Fabry disease: an international cohort study

Por Arends, Maarten, Biegstraaten, Marieke, Wanner, Christoph, Sirrs, Sandra, Mehta, Atul, Elliott, Perry M, Oder, Daniel, Watkinson, Oliver T, Bichet, Daniel G, Khan, Aneal, Iwanochko, Mark, Vaz, Frédéric M, van Kuilenburg, André B P, West, Michael L, Hughes, Derralynn A, Hollak, Carla E M
Publicado no J Med Genet (2018)

Lentivector Iterations and Pre-Clinical Scale-Up/Toxicity Testing: Targeting Mobilized CD34(+) Cells for Correction of Fabry Disease

Por Huang, Ju, Khan, Aneal, Au, Bryan C., Barber, Dwayne L., López-Vásquez, Lucía, Prokopishyn, Nicole L., Boutin, Michel, Rothe, Michael, Rip, Jack W., Abaoui, Mona, Nagree, Murtaza S., Dworski, Shaalee, Schambach, Axel, Keating, Armand, West, Michael L., Klassen, John, Turner, Patricia V., Sirrs, Sandra, Rupar, C. Anthony, Auray-Blais, Christiane, Foley, Ronan, Medin, Jeffrey A.
Publicado no Mol Ther Methods Clin Dev (2017)