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1

Does market exclusivity hinder the development of Follow-on Orphan Medicinal Products in Europe?

Anne EM Brabers, Ellen HM Moors, Sonja van Weely, Remco LA de Vrueh

Journal:

Orphanet Journal of Rare Diseases

Year:

2011

Language:

english

File:

PDF, 191 KB

english, 2011

2

A New Coding System for Metabolic Disorders Demonstrates Gaps in the International Disease Classifications ICD-10 and SNOMED-CT, Which Can Be Barriers to Genotype-Phenotype Data Sharing

Sollie, Annet, Sijmons, Rolf H., Lindhout, Dick, van der Ploeg, Ans T., Rubio Gozalbo, M. Estela, Smit, G. Peter A., Verheijen, Frans, Waterham, Hans R., van Weely, Sonja, Wijburg, Frits A., Wijburg,

Journal:

Year:

2013

Language:

english

File:

PDF, 331 KB

english, 2013

3

Chalcone isomerase in flowers of mutants of Petunia hybrida

S. van Weely, A. Bleumer, R. Spruyt and A.W. Schram

Journal:

Year:

1983

Language:

english

File:

PDF, 691 KB

english, 1983

4

Characterization of glucocerebrosidase in Greek Gaucher disease patients: mutation analysis and biochemical studies

H. Michelakakis, E. Dimitriou, S. Van Weely, R. G. Boot, I. Mavridou, M. Verhoek, J. M. F. G. Aerts

Journal:

Journal of Inherited Metabolic Disease

Year:

1995

Language:

english

File:

PDF, 395 KB

english, 1995

5

Predictors of orphan drug approval in the European Union

Harald E. Heemstra, Remco L. de Vrueh, Sonja van Weely, Hans A. Büller, Hubert G. M. Leufkens

Journal:

European Journal of Clinical Pharmacology

Year:

2008

Language:

english

File:

PDF, 158 KB

english, 2008

6

The frequency of lysosomal storage diseases in The Netherlands

B.J.H.M. Poorthuis, R.A. Wevers, W.J. Kleijer, J.E.M. Groener, J.G.N. de Jong, S. van Weely, K.E. Niezen-Koning, O.P. van Diggelen

Journal:

Year:

1999

Language:

english

File:

PDF, 47 KB

english, 1999

7

Oligosaccharide excretion in adult Gaucher disease

J. G. N. de Jong, J. M. F. G. Aerts, S. van Weely, C. E. M. Hollak, J. van Pelt, L. M. J. van Woerkom, M. L. F. Liebrand-van Sambeek, R. A. Wevers

Journal:

Journal of Inherited Metabolic Disease

Year:

1998

Language:

english

File:

PDF, 188 KB

english, 1998

8

Inhibition of substrate synthesis as a strategy for glycolipid lysosomal storage disease therapy

F.M. Platt, M. Jeyakumar, U. Andersson, D.A. Priestman, R.A. Dwek, T.D. Butters, T.M. Cox, R.H. Lachmann, C. Hollak, J.M.F.G. Aerts, S. Van Weely, M. Hrebícek, C. Moyses, I. Gow, D. Elstein, A. Zimran

Journal:

Journal of Inherited Metabolic Disease

Year:

2001

Language:

english

File:

PDF, 142 KB

english, 2001

9

Conditions affecting the activity of glucocerebrosidase purified fom spleens of control subjects and patients with type 1 Gaucher disease

Johannes M.F.G. Aerts, M.Clara Sa Miranda, Elidabeth M. Brouwer-Kelder, Sonja Van Weely, John A. Barranger, Joseph M. Tager

Journal:

Biochimica et Biophysica Acta (BBA) - Protein Structure and Molecular Enzymology

Year:

1990

Language:

english

File:

PDF, 852 KB

english, 1990

10

Orphan drug development across Europe: bottlenecks and opportunities

Harald E. Heemstra, Remco L.A. de Vrueh, Sonja van Weely, Hans A. Büller, Hubert G.M. Leufkens

Journal:

Drug Discovery Today

Year:

2008

Language:

english

File:

PDF, 377 KB

english, 2008

11

Translation of rare disease research into orphan drug development: disease matters

Harald E. Heemstra, Sonja van Weely, Hans A. Büller, Hubert G.M. Leufkens, Remco L.A. de Vrueh

Journal:

Drug Discovery Today

Year:

2009

Language:

english

File:

PDF, 399 KB

english, 2009

12

Serial chitotriosidase activity estimations in neonatal systemic candidiasis

J Labadaridis, E Dimitriou, C Costalos, J Aerts, S van Weely, WE Donker-Koopman, H Michelakakis

Journal:

Acta Paediatrica

Year:

1998

Language:

english

File:

PDF, 43 KB

english, 1998

13

Activity of glucocerebrosidase in extracts of different cell types from type 1 Gaucher disease patients

M. Clara Sa Miranda, Johannes M. F. G. Aerts, Rui Pinto, Augusta Fontes, Lucia Wanzeller de Lacerda, Sonja van Weely, John Barranger, Joseph M. Tager

Journal:

Clinical Genetics

Year:

1990

Language:

english

File:

PDF, 644 KB

english, 1990

14

Function of oligosaccharide modification in glucocerebrosidase, a membrane-associated lysosomal hydrolase

Sonja VAN WEELY, Johannes M. F. G. AERTS, Marinella B. VAN LEEUWEN, Judith C. HEIKOOP, Wilma E. DONKER-KOOPMAN, John A. BARRANGER, Joseph M. TAGER, André W. SCHRAM

Journal:

Year:

1990

Language:

english

File:

PDF, 1.13 MB

english, 1990

15

Quantitative Analysis of the Targeting of Mannose-Terminal Glucocerebrosidase : Predominant Uptake by Liver Endothelial Cells

Martin K. Bijsterbosch, Wilma Donker, Hendrika Van De Bilt, Sonja Van Weely, Theo J. C. Van Berkel, Johannes M. F. G. Aerts

Journal:

Year:

1996

Language:

english

File:

PDF, 703 KB

english, 1996

16

Making biodiversity meaningful through environmental education

Van Weelie, Daan, Wals, Arjen

Journal:

International Journal of Science Education

Year:

2002

Language:

english

File:

PDF, 201 KB

english, 2002

17

Low-Dose N-Butyldeoxynojirimycin (OGT 918) for Type I Gaucher Disease: Volume 28, Number 2 (2002), pages 127–133

Rene Heitner, Deborah Elstein, Johannes Aerts, Sonja van Weely, Ari Zimran

Journal:

Blood Cells, Molecules, and Diseases

Year:

2002

Language:

english

File:

PDF, 7 KB

english, 2002

18

Low-Dose N-Butyldeoxynojirimycin (OGT 918) for Type I Gaucher Disease

Rene Heitner, Deborah Elstein, Johannes Aerts, Sonja van Weely, Ari Zimran

Journal:

Blood Cells, Molecules, and Diseases

Year:

2002

Language:

english

File:

PDF, 68 KB

english, 2002

19

The identification of type 1 Gaucher disease patients, asymptomatic cases and carriers in The Netherlands using urine samples: An evaluation

J.M.F.G. Aerts, M.C.Sa Miranda, L.Wanzeller de Lacerda, S. van Weely, W. Donker-Koopman, B. Brouwer-Kelder, D.C. Jansen, M. van Leeuwen, A.W. Schram, A. Tsiapara, J.M. Tager

Journal:

Clinica Chimica Acta

Year:

1991

Language:

english

File:

PDF, 1.02 MB

english, 1991

20

Characterization of glucocerebrosidase in peripheral blood cells and cultured blastoid cells

J.M.F.G. Aerts, J. Heikoop, S. Van Weely, W.E. Donker-Koopman, J.A. Barranger, J.M. Tager, A.W. Schram

Journal:

Experimental Cell Research

Year:

1988

Language:

english

File:

PDF, 698 KB

english, 1988

21

Efficient routing of glucocerebrosidase to lysosomes requires complex oligosaccharide chain formation

J.M.F.G. Aerts, S. Brul, W.E. Donker-Koopman, S. van Weely, G.J. Murray, J.A. Barranger, J.M. Tager, A.W. Schram

Journal:

Biochemical and Biophysical Research Communications

Year:

1986

Language:

english

File:

PDF, 319 KB

english, 1986

22

Novel oral treatment of Gaucher's disease with N-butyldeoxynojirimycin (OGT 918) to decrease substrate biosynthesis

Timothy Cox, Robin Lachmann, Carla Hollak, Johannes Aerts, Sonja van Weely, Martin Hrebícek, Frances Platt, Terry Butters, Raymond Dwek, Chris Moyses, Irene Gow, Deborah Elstein, Ari Zimran

Journal:

Year:

2000

Language:

english

File:

PDF, 97 KB

english, 2000

23

The facile detection of 1505G→A in Gaucher patients with different phenotypes

Marina Moraitou, Sonja van Weely, Marri Verhoek, Johannes Aerts, Evangelia Dimitriou, Helen Michelakakis

Journal:

Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease

Year:

2001

Language:

english

File:

PDF, 344 KB

english, 2001

24

Plasma tumor necrosis factor-a (TNF-a) levels in Gaucher disease

Helen Michelakakis, Cleopatra Spanou, Anastasia Kondyli, Evagelia Dimitriou, Sonja Van Weely, Carla E.M. Hollak, Marinus H.J. Van Oers, Johannes M.F.G. Aerts

Journal:

Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease

Year:

1996

Language:

english

File:

PDF, 259 KB

english, 1996

25

Hepatosplenomegalic lipidosis: what unless Gaucher? Adult cholesteryl ester storage disease (CESD) with anemia, mesenteric lipodystrophy, increased plasma chitotriosidase activity and a homozygous lysosomal acid lipase −1 exon 8 splice junction mutation

Stephan vom Dahl, Klaus Harzer, Arndt Rolfs, Bettina Albrecht, Claus Niederau, Christoph Vogt, Sonja van Weely, Johannes Aerts, Gerd Müller, Dieter Häussinger

Journal:

Journal of Hepatology

Year:

1999

Language:

english

File:

PDF, 820 KB

english, 1999

26

Moving instead of asking? Performance-based tests and BASFI-questionnaire measure different aspects of physical function in ankylosing spondylitis

Salima FE van Weely, J Christiaan van Denderen…

Journal:

Arthritis Research & Therapy

Year:

2012

Language:

english

File:

PDF, 140 KB

english, 2012

27

Clinical phenotype of Gaucher disease in relation to properties of mutant glucocerebrosidase in cultured fibroblasts

Van Weely, Sonja, Van Leeuwen, Marinella B., Jansen, Ineke D.C., De Bruijn, Marianne A.C., Brouwer-Kelder, Elisabeth M., Schram, AndréW., Sa Miranda, M.Clara, Barranger, John A., Petersen, Evelyn M.,

Journal:

Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease

Year:

1991

Language:

english

File:

PDF, 1.02 MB

english, 1991

28

Demonstration of the existence of a second, non-lysosomal glucocerebrosidase that is not deficient in Gaucher disease

van Weely, Sonja, Brandsma, Margreet, Strijland, Anneke, Tager, Joseph M., Aerts, Johannes M.F.G.

Journal:

Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease

Year:

1993

Language:

english

File:

PDF, 742 KB

english, 1993

29

Glucocerebrosidase, a lysosomal enzyme that does not undergo oligosaccharide phosphorylation

Aerts, Johannes M.F.G., Schram, AndréW., Strijland, Anneke, van Weely, Sonja, Jonsson, Lisbeth M.V., Tager, Joseph M., Sorrell, Susan H., Ginns, Edward I., Barranger, John A., Murray, Gary J.

Journal:

Biochimica et Biophysica Acta (BBA) - General Subjects

Year:

1988

Language:

english

File:

PDF, 449 KB

english, 1988

30

What do we miss? ASAS non-responders on anti-TNF therapy show improvement in performance-based physical function

van Weely, S. F. E., van Denderen, J. C., Steultjens, M. P. M., Nurmohamed, M. T., Dijkmans, B. A. C., Dekker, J., van der Horst-Bruinsma, I. E.

Journal:

Year:

2013

Language:

english

File:

PDF, 156 KB

english, 2013

31

Objective Evaluation of Physical Functioning after Tumor Necrosis Factor Inhibitory Therapy in Patients with Ankylosing Spondylitis: A Selection of 3 Feasible Performance-based Tests

van Weely, S. F. E., Dekker, J., Steultjens, M. P. M., van Denderen, J. C., Nurmohamed, M. T., Dijkmans, B. A. C., van der Horst-Bruinsma, I. E.

Journal:

The Journal of Rheumatology

Year:

2015

Language:

english

File:

PDF, 243 KB

english, 2015

32

Reproducibility of performance measures of physical function based on the BASFI, in ankylosing spondylitis

van Weely, S. F. E., van Denderen, C. J., van der Horst-Bruinsma, I. E., Nurmohamed, M. T., Dijkmans, B. A. C., Dekker, J., Steultjens, M. P. M.

Journal:

Year:

2009

Language:

english

File:

PDF, 220 KB

english, 2009

33

Continuous improvement of physical functioning in Ankylosing Spondylitis patients by TNF inhibitors: 3-year follow-up and predictors

van Weely, Salima F.E., Kneepkens, Eva L., Nurmohamed, Mike T., Dekker, Joost, van der Horst-Bruinsma, Irene E.

Journal:

Arthritis Care & Research

Year:

2016

Language:

english

File:

PDF, 879 KB

english, 2016

34

Comparative study on glucocerebrosidase in spleens from patients with Gaucher disease

Aerts, J M F G, Donker-Koopman, W E, Brul, S, Van Weely, S, Sa Miranda, M C, Barranger, J A, Tager, J M, Schram, A W

Journal:

Biochemical Journal

Year:

1990

Language:

english

File:

PDF, 1.60 MB

english, 1990

35

The importance of international collaboration for rare diseases research—a European perspective

Julkowska, D, Austin, C P, Cutillo, C M, Gancberg, D, Hager, C, Halftermeyer, J, Jonker, A H, Lau, L P L, Norstedt, I, Rath, A, Schuster, R, Simelyte, E, van Weely, S

Journal:

Year:

2017

Language:

english

File:

PDF, 2.34 MB

english, 2017

36

Recontextualising biodiversity in school practice

van Weelie, Daan, Boersma, Kerst

Journal:

Journal of Biological Education

Year:

2017

Language:

english

File:

PDF, 853 KB

english, 2017

37

[BMJ Publishing Group Ltd and European League Against Rheumatism Annual European Congress of Rheumatology, 14–17 June, 2017 - ()] Poster Presentations - THU0748-HPR Recommendations on physical therapy prescription for axial spondyloarthritis in the netherlands

Weely, S van, Giesen, F van der, Lopuhaa, N, Gaalen, F van, Vlieland, T Vliet

Language:

english

File:

PDF, 65 KB

english

38

[BMJ Publishing Group Ltd and European League Against Rheumatism Annual European Congress of Rheumatology, 14–17 June, 2017 - ()] Poster Presentations - THU0730-HPR Content and supervision of group exercise therapy (GET) for axial spondyloarthritis (AXSPA) in the netherlands; a nation wide survey

Giesen, F Van Der, Weely, S Van, Lopuhaa, N, Vlieland, T Vliet

Language:

english

File:

PDF, 129 KB

english

39

AB0558 Three easy tests of physical functioning (aspi) show improvement after anti-tnf therapy, even in asas20 non-responders in ankylosing spondylitis

van Weely, S., Dekker, J., Steultjens, M., van Denderen, C., Nurmohamed, M., Dijkmans, B., van der Horst-Bruinsma, I.

Journal:

Annals of the Rheumatic Diseases

Year:

2013

Language:

english

File:

PDF, 218 KB

english, 2013

40

OP0208-HPR Three Year Course and Prediction of Physical Functioning in TNFI Treated as Patients

Van Weely, S., Kneepkens, E., Nurmohamed, M., Steultjens, M., Dekker, J., van der Horst-Bruinsma, I.

Journal:

Annals of the Rheumatic Diseases

Year:

2014

Language:

english

File:

PDF, 82 KB

english, 2014

41

Supervised Group Exercise in Axial Spondyloarthritis: Patients’ Satisfaction and Perspective on Evidence‐Based Enhancements

Hilberdink, Bas, van der Giesen, Florus, Vliet Vlieland, Thea, van Gaalen, Floris, van Weely, Salima

Journal:

Arthritis Care & Research

Year:

2019

Language:

english

File:

PDF, 258 KB

english, 2019

42

How to optimize exercise behavior in axial spondyloarthritis? Results of an intervention mapping study

Hilberdink, Bas, van der Giesen, Florus, Vliet Vlieland, Thea, Nijkamp, Marjan, van Weely, Salima

Journal:

Patient Education and Counseling

Year:

2019

File:

PDF, 2.56 MB

2019

43

Responsiveness and Interpretability of 2 Measures of Physical Function in Patients With Spondyloarthritis

Fongen, Camilla, Dagfinrud, Hanne, Bilberg, Annelie, Pedersen, Elisabeth, Johansen, Melissa Woll, van Weely, Salima, Hagen, Kåre Birger, Sveaas, Silje Halvorsen

Journal:

Physical Therapy

Year:

2020

Language:

english

File:

PDF, 958 KB

english, 2020

44

Adequately dosed aerobic physical activity in people with axial spondyloarthritis: associations with physical therapy

Hilberdink, Bas, Vlieland, Thea Vliet, van der Giesen, Florus, van Gaalen, Floris, Goekoop, Robbert, Peeters, Andreas, Fiocco, Marta, van Weely, Salima

Journal:

Rheumatology International

Year:

2020

File:

PDF, 776 KB

2020

45

[BMJ Publishing Group Ltd and European League Against Rheumatism Annual European Congress of Rheumatology, EULAR 2019, Madrid, 12â15 June 2019 - ()] Spondyloarthritis â treatment - FRI0393âPATIENTSâ SATISFACTION AND PREFERENCES TOWARDS SUPERVISED GROUP EXERCISE FOR PEOPLE WITH AXIAL SPONDYLOARTHRITIS

Hilberdink, Bas, Giesen, Florus van der, Vlieland, Thea Vliet, van Gaalen, Floris A., Weely, Salima van

Year:

2019

File:

PDF, 240 KB

2019

46

[BMJ Publishing Group Ltd and European League Against Rheumatism Annual European Congress of Rheumatology, EULAR 2019, Madrid, 12â15 June 2019 - ()] Poster Presentations - THU0367âANALYSING IMPAIRMENTS IN PHYSICAL PERFORMANCE (AS ASSESSED BY THE AS PERFORMANCE INDEX (ASPI)) IN PATIENTS WITH AXIAL SPONDYLOARTHRITIS

Ahomaa, Eerik, Kiltz, Uta, BÃ¼hring, BjÃ¶rn, Baraliakos, Xenofon, Weely, Salima van, Braun, Juergen

Year:

2019

File:

PDF, 145 KB

2019

47

[BMJ Publishing Group Ltd and European League Against Rheumatism Annual European Congress of Rheumatology, EULAR 2019, Madrid, 12â15 June 2019 - ()] Spondyloarthritis â treatment - FRI0376âDIFFERENCES IN PHYSICAL ACTIVITY BETWEEN AXIAL SPONDYLOARTHRITIS PATIENTS WITH AND WITHOUT PHYSICAL THERAPY

Hilberdink, Bas, Giesen, Florus van der, Vlieland, Thea Vliet, van Gaalen, Floris A., Ronday, Karel, Peeters, Andreas, Weely, Salima van

Year:

2019

File:

PDF, 176 KB

2019

48

Organisation and content of supervised group exercise for people with axial spondyloarthritis in The Netherlands

Hilberdink, Bas, van der Giesen, Florus, Vliet Vlieland, Thea, van Weely, Salima

Journal:

Rheumatology International

Year:

2020

File:

PDF, 597 KB

2020