Different diseases, different needs: Patient preferences for gene therapy in lysosomal storage disorders, a probabilistic threshold technique survey von Corazolla, Eleonore M, Eskes, Eline C B, Veldwijk, Jorien, Brands, Marion M M G, Dekker, Hanka, van de Mheen, Erica, Langeveld, Mirjam, Hollak, Carla E M, Sjouke, Barbara

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Patients' view on gene therapy development for lysosomal storage disorders: a qualitative study von Eskes, Eline C B, Beishuizen, Cathrien R L, Corazolla, Eleonore M, van Middelaar, Tessa, Brands, Marion M M G, Dekker, Hanka, van de Mheen, Erica, Langeveld, Mirjam, Hollak, Carla E M, Sjouke, Barbara

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

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