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19 p, 2.8 MB Consensus recommendations for the treatment and management of patients with Fabry disease on migalastat : a modified Delphi study / Bichet, Daniel G. (University of Montréal, Canada) ; Hopkin, Robert J. (University of Cincinnati College of Medicine, United States) ; Aguiar, Patrício (Lisbon University) ; Allam, Sridhar R. (Texas Christian University, Fort Worth, United States) ; Chien, Yin-Hsiu (National Taiwan University College of Medicine, Taipei, Taiwan) ; Giugliani, Roberto (Postgraduate Program in Genetics and Molecular Biology (PPGBM) at Federal University of Rio Grande do Sul (UFRGS), Porto Alegre, Brazil) ; Kallish, Staci (University of Pennsylvania, Philadelphia, United States) ; Kineen, Sabina (Patient Advocate, United States) ; Lidove, Olivier (French Network of Inherited Metabolic Disorders (G2m), France) ; Niu, Dau-Ming (National Yang Ming Chiao Tung University, Taipei, Taiwan) ; Olivotto, Iacopo (Meyer University Children's Hospital, Florence, Italy) ; Politei, Juan (Fundacion Para el Estudio de Enfermedades Neurometabolicas (FESEN), Buenos Aires, Argentina) ; Rakoski, Paul (Patient Advocate, United States) ; Torra Balcells, Roser (Institut d'Investigació Biomèdica Sant Pau) ; Tøndel, Camilla (University of Bergen, Norway) ; Hughes, Derralynn A (University College London) ; Universitat Autònoma de Barcelona. Departament de Medicina
Fabry disease is a progressive disorder caused by deficiency of the α-galactosidase A enzyme (α-Gal A), leading to multisystemic organ damage with heterogenous clinical presentation. The addition of the oral chaperone therapy migalastat to the available treatment options for Fabry disease is not yet universally reflected in all treatment guidelines. [...]
2023 - 10.3389/fmed.2023.1220637
Frontiers in Medicine, Vol. 10 (september 2023)  
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5 Allam, S.
1 Allam, S.D.
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