未登录Twenty years of the Fabry Outcome Survey (FOS): insights, achievements, and lessons learned from a global patient registry
Beck, Michael; Ramaswami, Uma; Hernberg-Stahl, Elizabeth; Hughes, Derralynn A.; Kampmann, Christoph; Mehta, Atul B.; Nicholls, Kathleen; Niu, Dau-Ming; Pintos-Morell, Guillem; Reisin, Ricardo; West, Michael L.; Schenk, Jorn; Anagnostopoulou, Christina; Botha, Jaco; Giugliani, Roberto
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收藏In-depth phenotyping for clinical stratification of Gaucher disease
D'Amore, Simona; Page, Kathleen; Donald, Aimee; Taiyari, Khadijeh; Tom, Brian; Deegan, Patrick; Tan, Chong Y.; Poole, Kenneth; Jones, Simon A.; Mehta, Atul; Hughes, Derralynn; Sharma, Reena; Lachmann, Robin H.; Chakrapani, Anupam; Geberhiwot, Tarekegn; Santra, Saikat; Banka, Siddarth; Cox, Timothy M.
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收藏Clinical outcomes after 4.5 years of eliglustat therapy for Gaucher disease type 1: Phase 3 ENGAGE trial final results
Mistry, Pramod K.; Lukina, Elena; Ben Turkia, Hadhami; Shankar, Suma P.; Feldman, Hagit Baris; Ghosn, Marwan; Mehta, Atul; Packman, Seymour; Lau, Heather; Petakov, Milan; Assouline, Sarit; Balwani, Manisha; Danda, Sumita; Hadjiev, Evgueniy; Ortega, Andres; Foster, Meredith C.; Gaemers, Sebastiaan J. M.; Peterschmitt, M. Judith
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收藏Assessment of plasma lyso-Gb3 for clinical monitoring of treatment response in migalastat-treated patients with Fabry disease (September, 10.1038/s41436-020-00968-z, 2020)
Bichet, Daniel G.; Aerts, Johannes M.; Auray-Blais, Christiane; Maruyama, Hiroki; Mehta, Atul B.; Skuban, Nina; Krusinska, Eva; Schiffmann, Raphael
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收藏Brain Microglial Activation Increased in Glucocerebrosidase (GBA) Mutation Carriers without Parkinson's disease
Mullin, Stephen; Stokholm, Morten Gersel; Hughes, Derralyn; Mehta, Atul; Parbo, Peter; Hinz, Rainer; Pavese, Nicola; Brooks, David J.; Schapira, Anthony H., V
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收藏Fatigue, quality of life and physical fitness following an exercise intervention in multiple myeloma survivors (MASCOT): an exploratory randomised Phase 2 trial utilising a modified Zelen design
Koutoukidis, Dimitrios A.; Land, Joanne; Hackshaw, Allan; Heinrich, Malgorzata; McCourt, Orla; Beeken, Rebecca J.; Philpott, Stephanie; DeSilva, Dunnya; Rismani, Ali; Rabin, Neil; Popat, Rakesh; Kyriakou, Charalampia; Papanikolaou, Xenofon; Mehta, Atul; Paton, Bruce; Fisher, Abigail; Yong, Kwee L.
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收藏Individual patient responses to eliglustat in treatment-naive adults with Gaucher disease type 1: Final data from the phase 3 ENGAGE trial
Mistry, Pramod K.; Lukina, Elena; Ben Turkia, Hadhami; Shankar, Suma; Feldman, Hagit Baris; Ghosn, Marwan; Mehta, Atul; Packman, Seymour; Lau, Heather; Petakov, Milan; Assouline, Sarit; Balwani, Manisha; Danda, Sumita; Hadjiev, Evgueniy; Ortega, Andres; Foster, Meredith C.; Gaemers, Sebastiaan J. M.; Peterschmitt, M. Judith
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收藏Proposed Stages of Myocardial Phenotype Development in Fabry Disease
Nordin, Sabrina; Kozor, Rebecca; Medina-Menacho, Katia; Abdel-Gadir, Amna; Baig, Shanat; Sado, Daniel M.; Lobascio, Ilaria; Murphy, Elaine; Lachmann, Robin H.; Mehta, Atul; Edwards, Nicola C.; Ramaswami, Uma; Steeds, Richard P.; Hughes, Derralynn; Moon, James C.
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收藏Evolution and clustering of prodromal parkinsonian features in GBA1 carriers
Mullin, Stephen; Beavan, Michelle; Bestwick, Jonathan; McNeill, Alisdair; Proukakis, Christos; Cox, Timothy; Hughes, Derralynn; Mehta, Atul; Zetterberg, Henrik; Schapira, Anthony H. V.
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