Articles with public access mandates - C.E.M. HollakLearn more
Not available anywhere: 1
Liposome-targeted recombinant human acid sphingomyelinase: Production, formulation, and in vitro evaluation
MH Aldosari, RP de Vries, LR Rodriguez, NA Hesen, N Beztsinna, ...
European Journal of Pharmaceutics and Biopharmaceutics 137, 185-195, 2019
Mandates: Netherlands Organisation for Scientific Research
Available somewhere: 39
Recommendations for initiation and cessation of enzyme replacement therapy in patients with Fabry disease: the European Fabry Working Group consensus document
M Biegstraaten, R Arngrímsson, F Barbey, L Boks, F Cecchi, PB Deegan, ...
Orphanet journal of rare diseases 10, 1-10, 2015
Mandates: Netherlands Organisation for Health Research and Development
Long term enzyme replacement therapy for Fabry disease: effectiveness on kidney, heart and brain
SM Rombach, BE Smid, MG Bouwman, GE Linthorst, MGW Dijkgraaf, ...
Orphanet journal of rare diseases 8, 1-9, 2013
Mandates: Netherlands Organisation for Health Research and Development
Screening, diagnosis, and management of patients with Fabry disease: conclusions from a “Kidney Disease: Improving Global Outcomes”(KDIGO) Controversies Conference
R Schiffmann, DA Hughes, GE Linthorst, A Ortiz, E Svarstad, DG Warnock, ...
Kidney international 91 (2), 284-293, 2017
Mandates: US National Institutes of Health
Outrageous prices of orphan drugs: a call for collaboration
L Luzzatto, HI Hyry, A Schieppati, E Costa, S Simoens, F Schaefer, ...
The Lancet 392 (10149), 791-794, 2018
Mandates: Research Foundation (Flanders)
The Morquio A Clinical Assessment Program: baseline results illustrating progressive, multisystemic clinical impairments in Morquio A subjects
P Harmatz, KE Mengel, R Giugliani, V Valayannopoulos, SP Lin, R Parini, ...
Molecular Genetics and Metabolism 109 (1), 54-61, 2013
Mandates: US National Institutes of Health
Enzyme replacement and substrate reduction therapy for Gaucher disease
E Shemesh, L Deroma, B Bembi, P Deegan, C Hollak, NJ Weinreb, ...
Cochrane Database of Systematic Reviews, 2015
Mandates: UK Medical Research Council, National Institute for Health Research, UK
Management goals for type 1 Gaucher disease: An expert consensus document from the European working group on Gaucher disease
M Biegstraaten, TM Cox, N Belmatoug, MG Berger, T Collin-Histed, ...
Blood Cells, Molecules, and Diseases 68, 203-208, 2018
Mandates: UK Medical Research Council, Netherlands Organisation for Health Research …
Dose-response relationships for enzyme replacement therapy with imiglucerase/alglucerase in patients with Gaucher disease type 1
GA Grabowski, K Kacena, JA Cole, CEM Hollak, L Zhang, J Yee, ...
Genetics in Medicine 11 (2), 92-100, 2009
Mandates: US National Institutes of Health
Agalsidase alfa versus agalsidase beta for the treatment of Fabry disease: an international cohort study
M Arends, M Biegstraaten, C Wanner, S Sirrs, A Mehta, PM Elliott, D Oder, ...
Journal of Medical Genetics 55 (5), 351-358, 2018
Mandates: Netherlands Organisation for Health Research and Development, Federal …
Retrospective study of long-term outcomes of enzyme replacement therapy in Fabry disease: analysis of prognostic factors
M Arends, M Biegstraaten, DA Hughes, A Mehta, PM Elliott, D Oder, ...
PloS one 12 (8), e0182379, 2017
Mandates: Netherlands Organisation for Health Research and Development, Federal …
Quality of life in patients with Fabry disease: a systematic review of the literature
M Arends, CEM Hollak, M Biegstraaten
Orphanet journal of rare diseases 10, 1-10, 2015
Mandates: Netherlands Organisation for Health Research and Development
Bone health in phenylketonuria: a systematic review and meta-analysis
S Demirdas, KE Coakley, PH Bisschop, CEM Hollak, AM Bosch, ...
Orphanet journal of rare diseases 10, 1-17, 2015
Mandates: US National Institutes of Health
Establishing 3-nitrotyrosine as a biomarker for the vasculopathy of Fabry disease
L Shu, A Vivekanandan-Giri, S Pennathur, BE Smid, JMFG Aerts, ...
Kidney International 86 (1), 58-66, 2014
Mandates: US National Institutes of Health
Cost-effectiveness of enzyme replacement therapy for Fabry disease
SM Rombach, CEM Hollak, GE Linthorst, MGW Dijkgraaf
Orphanet journal of rare diseases 8, 1-9, 2013
Mandates: Netherlands Organisation for Health Research and Development
A monozygotic twin pair with highly discordant Gaucher phenotypes
M Biegstraaten, IN Van Schaik, J Aerts, M Langeveld, M Mannens, ...
Blood Cells, Molecules, and Diseases 46 (1), 39-41, 2011
Mandates: US National Institutes of Health
Chronic kidney disease and an uncertain diagnosis of Fabry disease: approach to a correct diagnosis
L van der Tol, E Svarstad, A Ortiz, C Tøndel, JP Oliveira, L Vogt, S Waldek, ...
Molecular Genetics and Metabolism 114 (2), 242-247, 2015
Mandates: National Institute for Health Research, UK, Government of Spain
Short‐term effect of estrogen on human bone marrow fat
EJ Limonard, AG Veldhuis‐Vlug, L van Dussen, JH Runge, MW Tanck, ...
Journal of Bone and Mineral Research 30 (11), 2058-2066, 2015
Mandates: Netherlands Organisation for Health Research and Development
The role of the clinician in the multi-omics era: are you ready?
CDM van Karnebeek, SB Wortmann, M Tarailo-Graovac, M Langeveld, ...
Journal of Inherited Metabolic Disease 41, 571-582, 2018
Mandates: Michael Smith Foundation for Health Research
Favourable effect of early versus late start of enzyme replacement therapy on plasma globotriaosylsphingosine levels in men with classical Fabry disease
M Arends, FA Wijburg, C Wanner, FM Vaz, ABP van Kuilenburg, ...
Molecular Genetics and Metabolism 121 (2), 157-161, 2017
Mandates: Netherlands Organisation for Health Research and Development
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