Concepedia

Standardising clinical outcomes measures for adult clinical trials in Fabry disease: A global Delphi consensus

David Moreno-Martínez, Patrício Aguiar, Christiane Auray‐Blais, Michael Beck, Daniel G. Bichet, Alessandro P. Burlina, David E. C. Cole, Perry Elliott, Ulla Feldt‐Rasmussen, Sandro Feriozzi,

Molecular Genetics and Metabolism · 2021 · 15 citations · 41 references

DOIFull text

Open access

Concepts

References

41

Clinical recommendations for cardiovascular magnetic resonance mapping of T1, T2, T2* and extracellular volume: A consensus statement by the Society for Cardiovascular Magnetic Resonance (SCMR) endorsed by the European Association for Cardiovascular Imaging (EACVI)

Daniel Messroghli, James Moon, Vanessa M. Ferreira et al. · Journal of Cardiovascular Magnetic Resonance · 2016 · 1.7K citations · Full text

, , +17

Safety and Efficacy of Recombinant Human α-Galactosidase A Replacement Therapy in Fabry's Disease

Christine M. Eng, Nathalie Guffon, William R. Wilcox et al. · New England Journal of Medicine · 2001 · 1.5K citations · Full text

, , +14

Enzyme Replacement Therapy in Fabry Disease

Raphael Schiffmann, Jeffrey B. Kopp, Howard A. Austin et al. · JAMA · 2001 · 1.3K citations

, , +13

Agalsidase-Beta Therapy for Advanced Fabry Disease

Maryam Banikazemi, Jan Bultas, Stephen Waldek et al. · Annals of Internal Medicine · 2007 · 566 citations

, , +7

Treatment of Fabry’s Disease with the Pharmacologic Chaperone Migalastat

Dominique P. Germain, Derralynn Hughes, Daniel G. Bichet et al. · New England Journal of Medicine · 2016 · 556 citations · Full text

, , +17