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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Acid sphingomyelinase deficiency and Gaucher disease in adults: Similarities and differences in two macrophage storage disorders von Eskes, Eline C. B., Dussen, Laura, Aerts, Johannes M. F. G., Lienden, Martijn J. C., Maas, Mario, Akkerman, Erik M., Kuilenburg, André B. P., Sjouke, Barbara, Hollak, Carla E. M.

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Renal involvement in a patient with the chronic visceral subtype of acid sphingomyelinase deficiency resembles Fabry disease von Eskes, Eline C. B., Lienden, Martijn J. C., Roelofs, Joris J. T. H., Vogt, Liffert, Aerts, Johannes M. F. G., Aten, Jan, Hollak, Carla E. M.

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Glycoprotein non-metastatic protein B (GPNMB) plasma values in patients with chronic visceral acid sphingomyelinase deficiency von Eskes, Eline C.B., van der Lienden, Martijn J.C., Sjouke, Barbara, van Vliet, Laura, Brands, Marion M.M.G., Hollak, Carla E.M., Aerts, Johannes M.F.G.

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Biochemical and imaging parameters in acid sphingomyelinase deficiency: Potential utility as biomarkers von Eskes, Eline C.B., Sjouke, Barbara, Vaz, Frédéric M., Goorden, Susan M.I., van Kuilenburg, André B.P., Aerts, Johannes M.F.G., Hollak, Carla E.M.

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Natural disease course of chronic visceral acid sphingomyelinase deficiency in adults: A first step toward treatment criteria von Eskes, Eline C B, van Dussen, Laura, Brands, Marion M M G, Vaz, Frédéric M, Aerts, Johannes M F G, van Kuilenburg, André B P, Sjouke, Barbara, Hollak, Carla E M

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Renal involvement in a patient with the chronic visceral subtype of acid sphingomyelinase deficiency resembles Fabry disease von Eskes, Eline C B, van der Lienden, Martijn J C, Roelofs, Joris J T H, Vogt, Liffert, Aerts, Johannes M F G, Aten, Jan, Hollak, Carla E M

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