Orphanet Journal of Rare Diseases | Home page

Orphanet Journal of Rare Diseases | Home page

Orphanet Journal of Rare Diseases

The following new articles have just been published in Orphanet Journal of Rare Diseases RESEARCH Migalastat improves diarrhea in patients with Fabry disease: clinical-biomarker correlations from the phase 3 FACETS trial Raphael Schiffmann, Daniel G. Bichet, Ana Jovanovic, Derralynn A. Hughes, Roberto Giugliani, Ulla Feldt-Rasmussen, Suma P. Shankar, Laura Barisoni, Robert B. Colvin, J. Charles Jennette, Fred Holdbrook, Andrew Mulberg, Jeffrey P. Castelli, Nina Skuban, Jay A. Barth and Kathleen Nicholls Orphanet Journal of Rare Diseases 2018, 13:68 | Published on: 27 April 2018 Full Text | PDF POSITION STATEMENT European principles of inhibitor management in patients with haemophilia P. L. F. Giangrande, C. Hermans, B. O’Mahony, P. de Kleijn, M. Bedford, A. Batorova, J. Blatný and K. Jansone Orphanet Journal of Rare Diseases 2018, 13:66 | Published on: 27 April 2018 Full Text | PDF RESEARCH Revealed preferences towards the appraisal of orphan drugs in Poland - multi criteria decision analysis Katarzyna Kolasa, Krzysztof Miroslaw Zwolinski, Vladimir Zah, Zoltán Kaló and Tadeusz Lewandowski Orphanet Journal of Rare Diseases 2018, 13:67 | Published on: 27 April 2018 Full Text | PDF

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