Quality of life in patients with Fabry disease: a systematic review of the literature

por Arends, Maarten, Hollak, Carla E. M., Biegstraaten, Marieke
Publicado 2015

Cost-effectiveness of enzyme replacement therapy for type 1 Gaucher disease

por van Dussen, Laura, Biegstraaten, Marieke, Hollak, Carla EM, Dijkgraaf, Marcel GW
Publicado 2014

Modelling Gaucher disease progression: long-term enzyme replacement therapy reduces the incidence of splenectomy and bone complications

por van Dussen, Laura, Biegstraaten, Marieke, Dijkgraaf, Marcel GW, Hollak, Carla EM
Publicado 2014

Autonomic neuropathy in Fabry disease: a prospective study using the Autonomic Symptom Profile and cardiovascular autonomic function tests

por Biegstraaten, Marieke, van Schaik, Ivo N, Wieling, Wouter, Wijburg, Frits A, Hollak, Carla EM
Publicado 2010

Poikilothermia in a 38-year-old Fabry patient

por Biegstraaten, Marieke, van Schaik, Ivo N., Hollak, Carla E. M., Wieling, Wouter, Linthorst, Gabor E.
Publicado 2010

Phenotype, disease severity and pain are major determinants of quality of life in Fabry disease: results from a large multicenter cohort study

por Arends, Maarten, Körver, Simon, Hughes, Derralynn A., Mehta, Atul, Hollak, Carla E. M., Biegstraaten, Marieke
Publicado 2017

Oncologic orphan drugs approved in the EU – do clinical trial data correspond with real-world effectiveness?

por Schuller, Yvonne, Biegstraaten, Marieke, Hollak, Carla E. M., Klümpen, Heinz-Josef, Gispen-de Wied, Christine C., Stoyanova-Beninska, Violeta
Publicado 2018

Retrospective study of long-term outcomes of enzyme replacement therapy in Fabry disease: Analysis of prognostic factors

por Arends, Maarten, Biegstraaten, Marieke, Hughes, Derralynn A., Mehta, Atul, Elliott, Perry M., Oder, Daniel, Watkinson, Oliver T., Vaz, Frédéric M., van Kuilenburg, André B. P., Wanner, Christoph, Hollak, Carla E. M.
Publicado 2017

The cognitive profile of type 1 Gaucher disease patients

por Biegstraaten, Marieke, Wesnes, Keith A., Luzy, Cécile, Petakov, Milan, Mrsic, Mirando, Niederau, Claus, Giraldo, Pilar, Hughes, Derralynn, Mehta, Atul, Mengel, Karl-Eugen, Hollak, Carla E. M, Maródi, László, van Schaik, Ivo N.
Publicado 2012

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 2018

Position statement on the role of healthcare professionals, patient organizations and industry in European Reference Networks

por Hollak, Carla E. M., Biegstraaten, Marieke, Baumgartner, Matthias R., Belmatoug, Nadia, Bembi, Bruno, Bosch, Annet, Brouwers, Martijn, Dekker, Hanka, Dobbelaere, Dries, Engelen, Marc, Groenendijk, Marike C., Lachmann, Robin, Langendonk, Janneke G., Langeveld, Mirjam, Linthorst, Gabor, Morava, Eva, Poll-The, Bwee Tien, Rahman, Shamima, Rubio-Gozalbo, M. Estela, Spiekerkoetter, Ute, Treacy, Eileen, Wanders, Ronald, Zschocke, Johannes, Hagendijk, Rob
Publicado 2016

Recommendations for initiation and cessation of enzyme replacement therapy in patients with Fabry disease: the European Fabry Working Group consensus document

por Biegstraaten, Marieke, Arngrímsson, Reynir, Barbey, Frederic, Boks, Lut, Cecchi, Franco, Deegan, Patrick B, Feldt-Rasmussen, Ulla, Geberhiwot, Tarekegn, Germain, Dominique P, Hendriksz, Chris, Hughes, Derralynn A, Kantola, Ilkka, Karabul, Nesrin, Lavery, Christine, Linthorst, Gabor E, Mehta, Atul, van de Mheen, Erica, Oliveira, João P, Parini, Rossella, Ramaswami, Uma, Rudnicki, Michael, Serra, Andreas, Sommer, Claudia, Sunder-Plassmann, Gere, Svarstad, Einar, Sweeb, Annelies, Terryn, Wim, Tylki-Szymanska, Anna, Tøndel, Camilla, Vujkovac, Bojan, Weidemann, Frank, Wijburg, Frits A, Woolfson, Peter, Hollak, Carla EM
Publicado 2015