E. Vroom

The patient’s view on rare disease trial design – a qualitative study

Charlotte M. W. Gaasterland, M. C. Jansen – van der Weide, M. J. du Prie – Olthof et al. · Orphanet Journal of Rare Diseases · 2019 · 70 citations · Full text

Public Health, Biostatistics, Clinical Epidemiology +10

Forty-Five Years of Duchenne Muscular Dystrophy in The Netherlands

J.C. van den Bergen, H.B. Ginjaar, A.J. van Essen et al. · Journal of Neuromuscular Diseases · 2014 · 52 citations · Full text

Medicine, Internal Medicine, Neurology +12

Transition in Duchenne Muscular Dystrophy: An expert meeting report and description of transition needs in an emergent patient population

D.G.M. Schrans, David Abbott, Holly L. Peay et al. · Neuromuscular Disorders · 2012 · 39 citations

Medicine, Neurology, Transitional Care +6

249th ENMC International Workshop: The role of brain dystrophin in muscular dystrophy: Implications for clinical care and translational research, Hoofddorp, The Netherlands, November 29th–December 1st 2019

J. Hendriksen, Mathula Thangarajh, Hermien E. Kan et al. · Neuromuscular Disorders · 2020 · 32 citations · Full text

Medicine, Neurology, Neuroscience +15

The POWER-tool: Recommendations for involving patient representatives in choosing relevant outcome measures during rare disease clinical trial design

Charlotte M. W. Gaasterland, Marijke C. Jansen‐van der Weide, E. Vroom et al. · Health Policy · 2018 · 17 citations · Full text

Pharmacy, Biostatistics, Evidence-based Practice +16