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Original Research

An open-label clinical trial of agalsidase alfa enzyme replacement therapy in children with Fabry disease who are naïve to enzyme replacement therapy

Ozlem Goker-Alpan1 Lysosomal Disorders Unit, Fairfax, VA, USACorrespondence[email protected]

Nicola Longo2 University of Utah, Salt Lake City, UT, USA

Marie McDonald3 Department of Pediatrics, Duke University, Durham, NC, USA

Suma P Shankar4 Department of Ophthalmology, Emory University School of Medicine, Atlanta, GA, USA;5 Department of Human Genetics, Emory University School of Medicine, Atlanta, GA, USA

Raphael Schiffmann6 Institute of Metabolic Disease, Baylor Research Institute, Dallas, TX, USA

Peter Chang7 Shire, Lexington, MA, USA

Yinghua Shen7 Shire, Lexington, MA, USA

Arian Pano7 Shire, Lexington, MA, USA

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Pages 1771-1781 | Published online: 25 May 2016

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Lina Palaiodimou, Panagiotis Kokotis, Christina Zompola, Georgia Papagiannopoulou, Eleni Bakola, Marianna Papadopoulou, Vasiliki Zouvelou, Dimitrios Petras, Charalampos Vlachopoulos & Georgios Tsivgoulis. (2023) Fabry Disease: Current and Novel Therapeutic Strategies. A Narrative Review. Current Neuropharmacology 21:3, pages 440-456.
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Lisa Bashorum, Gerard McCaughey, Owen Evans, Ashley C. Humphries, Richard Perry & Alasdair MacCulloch. (2022) Burden associated with Fabry disease and its treatment in 12–15 year olds: results from a European survey. Orphanet Journal of Rare Diseases 17:1.
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Moritz C. Halfmann, Sebastian Altmann, U. Joseph Schoepf, Constantin Reichardt, Julia B. Hennermann, Karl-Friedrich Kreitner, Roman Kloeckner, Felix Hahn, Christoph Dueber, Akos Varga-Szemes, Christoph Kampmann & Tilman Emrich. (2022) Left atrial strain correlates with severity of cardiac involvement in Anderson-Fabry disease. European Radiology 33:3, pages 2039-2051.
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Maria Helena Vaisbich, Luís Gustavo Modelli de Andrade, Cassiano Augusto Braga Silva & Fellype de Carvalho Barreto. (2022) Recommendations for the diagnosis and management of Fabry disease in pediatric patients: a document from the Rare Diseases Committee of the Brazilian Society of Nephrology (Comdora-SBN). Brazilian Journal of Nephrology 44:2, pages 268-280.
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Aneal Khan, Sandra M. Sirrs, Daniel G. Bichet, Chantal F. Morel, Adina Tocoian, Lan Lan & Michael L. West. (2021) The Safety of Agalsidase Alfa Enzyme Replacement Therapy in Canadian Patients with Fabry Disease Following Implementation of a Bioreactor Process. Drugs in R&D 21:4, pages 385-397.
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Angela Sun, Irene J. Chang, Christina Lam & Gerard T. Berry. 2021. Emery and Rimoin's Principles and Practice of Medical Genetics and Genomics. Emery and Rimoin's Principles and Practice of Medical Genetics and Genomics 563 682 .

Derralynn A Hughes, Patricio Aguiar, Patrick B Deegan, Fatih Ezgu, Andrea Frustaci, Olivier Lidove, Aleš Linhart, Jean-Claude Lubanda, James C Moon, Kathleen Nicholls, Dau-Ming Niu, Albina Nowak, Uma Ramaswami, Ricardo Reisin, Paula Rozenfeld, Raphael Schiffmann, Einar Svarstad, Mark Thomas, Roser Torra, Bojan Vujkovac, David G Warnock, Michael L West, Jack Johnson, Mark J Rolfe & Sandro Feriozzi. (2020) Early indicators of disease progression in Fabry disease that may indicate the need for disease-specific treatment initiation: findings from the opinion-based PREDICT-FD modified Delphi consensus initiative. BMJ Open 10:10, pages e035182.
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Aleš Linhart, Dominique P. Germain, Iacopo Olivotto, Mohammed M. Akhtar, Aris Anastasakis, Derralynn Hughes, Mehdi Namdar, Maurizio Pieroni, Albert Hagège, Franco Cecchi, Juan R. Gimeno, Giuseppe Limongelli & Perry Elliott. (2020) An expert consensus document on the management of cardiovascular manifestations of Fabry disease. European Journal of Heart Failure 22:7, pages 1076-1096.
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Yoo Jin Hong & Young Jin Kim. (2020) The Role of Cardiac MRI in the Diagnosis of Fabry Disease. Journal of the Korean Society of Radiology 81:2, pages 302.
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Dominique P. Germain, Alain Fouilhoux, Stéphane Decramer, Marine Tardieu, Pascal Pillet, Marc Fila, Serge Rivera, Georges Deschênes & Didier Lacombe. (2019) Consensus recommendations for diagnosis, management and treatment of Fabry disease in paediatric patients. Clinical Genetics 96:2, pages 107-117.
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Julia B. Hennermann, Laila Arash‐Kaps, György Fekete, Andreas Schaaf, Andreas Busch & Thomas Frischmuth. (2019) Pharmacokinetics, pharmacodynamics, and safety of moss‐aGalactosidase A in patients with Fabry disease. Journal of Inherited Metabolic Disease 42:3, pages 527-533.
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Marco Spada, Ralf Baron, Perry M. Elliott, Bruno Falissard, Max J. Hilz, Lorenzo Monserrat, Camilla Tøndel, Anna Tylki-Szymańska, Christoph Wanner & Dominique P. Germain. (2019) The effect of enzyme replacement therapy on clinical outcomes in paediatric patients with Fabry disease – A systematic literature review by a European panel of experts. Molecular Genetics and Metabolism 126:3, pages 212-223.
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Carlota Pascoal, Sandra Brasil, Rita Francisco, Dorinda Marques-da-Silva, Agnes Rafalko, Jaak Jaeken, Paula A. Videira, Luísa Barros & Vanessa dos Reis Ferreira. (2018) Patient and observer reported outcome measures to evaluate health-related quality of life in inherited metabolic diseases: a scoping review. Orphanet Journal of Rare Diseases 13:1.
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Hideto Morimoto, Yae Ito, Eiji Yoden, Masato Horie, Noboru Tanaka, Yoshikazu Komurasaki, Ryuji Yamamoto, Kazutoshi Mihara, Kohtaro Minami & Tohru Hirato. (2018) Non-clinical evaluation of JR-051 as a biosimilar to agalsidase beta for the treatment of Fabry disease. Molecular Genetics and Metabolism 125:1-2, pages 153-160.
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Christoph Wanner, Michael Arad, Ralf Baron, Alessandro Burlina, Perry M. Elliott, Ulla Feldt-Rasmussen, Victor V. Fomin, Dominique P. Germain, Derralynn A. Hughes, Ana Jovanovic, Ilkka Kantola, Aleš Linhart, Renzo Mignani, Lorenzo Monserrat, Mehdi Namdar, Albina Nowak, João-Paulo Oliveira, Alberto Ortiz, Maurizio Pieroni, Marco Spada, Anna Tylki-Szymańska, Camilla Tøndel, Miguel Viana-Baptista, Frank Weidemann & Max J. Hilz. (2018) European expert consensus statement on therapeutic goals in Fabry disease. Molecular Genetics and Metabolism 124:3, pages 189-203.
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An open-label clinical trial of agalsidase alfa enzyme replacement therapy in children with Fabry disease who are naïve to enzyme replacement therapy

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An open-label clinical trial of agalsidase alfa enzyme replacement therapy in children with Fabry disease who are naïve to enzyme replacement therapy

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