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Operational description of rare diseases: a reference to improve the recognition and visibility of rare diseases 罕见病的操作描述: 提高罕见病的识别和知名度的参考 Wang, Chiuhui Mary; Whiting, Amy Heagle; Rath, Ana; Anido, Roberta; Ardigo, Diego; Baynam, Gareth; Dawkins, Hugh; Hamosh, Ada; Le Cam, Yann; Malherbe, Helen; Molster, Caron M.; Monaco, Lucia; Padilla, Carmencita D.; Pariser, Anne R.; Robinson, Peter N.; Rodwell, Charlotte; Schaefer, Franz; Weber, Stefanie; Macchia, Flaminia 分享 收藏
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Global treatment response analysis of velmanase alfa long term enzyme replacement therapy for alpha-mannosidosis shows treatment benefit across ages Harmatz, Paul; Cattaneo, Federica; Ardigo, Diego; Geraci, Silvia; Muschol, Nicole; Amraoui, Yasmina; Gil-Campos, Mercedes; Tylki-Szymanska, Anna; Wijburg, Frits; De Meirleir, Linda; Van den Hout, Johanna M. P.; Dali, Christine I.; Jones, Simon A.; Lund, Allan M.; Borgwardt, Line; Guffon, Nathalie 分享 收藏
Global treatment responder analysis demonstrates clinically relevant effect of velmanase alfa long term enzyme replacement therapy for alpha mannosidosis, in a phase III randomized placebo controlled trial Harmatz, Paul; Cattaneo, Federica; Ardigo, Diego; Geraci, Silvia; Amraoui, Yasmina; Gil-Campos, Mercedes; De Meirleir, Linda; Muschol, Nicole; Tylki-Szymanska, Anna; Wijburg, Frits; Van den Hout, Johanna M. P.; Guffon, Nathalie; Dali, Christine I.; Jones, Simon A.; Lund, Allan M.; Borgwardt, Line 分享 收藏
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Future of Rare Diseases Research 2017-2027: An IRDiRC Perspective Austin, Christopher P.; Cutillo, Christine M.; Lau, Lilian P. L.; Jonker, Anneliene H.; Rath, Ana; Julkowska, Daria; Thomson, David; Terry, Sharon F.; de Montleau, Beatrice; Ardigo, Diego; Hivert, Virginie; Boycott, Kym M.; Baynam, Gareth; Kaufmann, Petra; Taruscio, Domenica; Lochmuller, Hanns; Suematsu, Makoto; Incerti, Carlo; Draghia-Akli, Ruxandra; Norstedt, Irene; Wang, Lu; Dawkins, Hugh J. S. 分享 收藏
Improvement in fine and gross motor proficiency after long-term enzyme replacement therapy with velmanase alfa (human recombinant alpha mannosidase) in alpha-mannosidosis patients Borgwardt, Line; Lund, Allan M.; Amraoui, Yasmine; Andersen, Oluf; De Meirleir, Linda; Dolhem, Philippe; Campos, Mercedes Gil; Guffon, Nathalie; Heron, Benedicte; Jameson, Elisabeth; Jones, Simon A.; Laroche, Cecile; Lindberg, Christoffer; Marquardt, Thorsten; Mengel, Karl-Eugene; Muschol, Nicole; Tylki-Szymanska, Anna; Van den Hout, Johanna M. P.; Van der Ploeg, Ans; Welling, Lindsey; Wijburg, Frits; Fogh, Jens; Geraci, Silvia; Ardigo, Diego; Cattaneo, Federica; Phillips, Dawn 分享 收藏
Long-term enzyme replacement therapy with velmanase alfa (human recombinant alpha-mannosidase) slows disease progression in adult patients suffering from alpha-mannosidosis Borgwardt, Line; Lund, Allan M.; Amraoui, Yasmina; Andersen, Oluf; De Meirleird, Linda; Dolhem, Philippe; Campos, Mercedes Gil; Guffon, Nathalie; Heron, Benedicte; Laroche, Cecile; Lindberg, Christoffer; Marquardt, Thorsten; Mengel, Karl-Eugene; Muschol, Nicole; Tylki-Szymanska, Anna; Van Den Hout, Johanna M. P.; Van Der Ploeg, Ans; Welling, Lindsey; Wijburg, Frits; Cole, Duncan; Fogh, Jens; Geraci, Silvia; Ardigo, Diego; Cattaneo, Federica 分享 收藏
Long-term enzyme replacement therapy with velmanase alfa (human recombinant alpha-mannosidase) improves mobility in alpha-mannosidosis patients Lund, Allan M.; Borgwardt, Line; Amraoui, Yasmina; Andersen, Oluf; De Meirleir, Linda; Dolhem, Philippe; Gil Campos, Mercedes; Guffon, Nathalie; Heron, Benedicte; Jameson, Elisabeth; Jones, Simon A.; Laroche, Cecile; Lindberg, Christoffer; Marquardt, Thorsten; Mengel, Karl-Eugene; Muschol, Nicole; Tylki-Szymanska, Anna; Van den Hout, Johanna M. P.; Van der Ploeg, Ans; Welling, Lindsey; Wijburg, Frits; Fogh, Jens; Geraci, Silvia; Ardigo, Diego; Cattaneo, Federica 分享 收藏
Improvement in pulmonary function and serum immunoglobulin G in long-term enzyme replacement therapy with velmanase alfa (human recombinant alpha-mannosidase) in alpha-mannosidosis patients Borgwardt, Line; Lund, Allan M.; De Meirleir, Linda; Amraoui, Yasmina; Andersen, Oluf; Dolhem, Philippe; Campos, Mercedes Gil; Guffon, Nathalie; Heron, Benedicte; Jameson, Elisabeth; Jones, Simon A.; Laroche, Cecile; Lindberg, Christoffer; Marquardt, Thorsten; Mengel, Karl-Eugene; Muschol, Nicole; Van den Hout, Johanna M. P.; Welling, Lindsey; Wijburg, Frits; Cole, Duncan; Van der Ploeg, Ans; Tylki-Szymanska, Anna; Fogh, Jens; Geraci, Silvia; Ardigo, Diego; Cattaneo, Federica 分享 收藏
COST-EFFECTIVENESS ANALYSIS OF EX-VIVO EXPANDED AUTOLOGOUS CORNEAL EPITHELIAL CELLS CONTAINING STEM CELLS TO REPAIR THE DAMAGED OCULAR SURFACE IN PATIENTS WITH MODERATE TO SEVERE LIMBAL STEM CELL DEFICIENCY DUE TO OCULAR BURNS IN THE UK Fordham, R.; Ciminata, G.; Madoni, A.; Magni, T.; Ardigo, D.; Pelosi, D.; Withe, M.; Sarnelli, V; Deltetto, I; Domini, B. 分享 收藏
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