Long term clinical history of an Italian cohort of infantile onset Pompe disease treated with enzyme replacement therapy

被引：0
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作者：
Rossella Parini
Paola De Lorenzo
Andrea Dardis
Alberto Burlina
Alessandra Cassio
Paolo Cavarzere
Daniela Concolino
Roberto Della Casa
Federica Deodato
Maria Alice Donati
Agata Fiumara
Serena Gasperini
Francesca Menni
Veronica Pagliardini
Michele Sacchini
Marco Spada
Roberta Taurisano
Maria Grazia Valsecchi
Maja Di Rocco
Bruno Bembi
机构：
[1] Pediatric Rare Diseases Unit,Centre of Biostatistics for Clinical Epidemiology, School of Medicine and Surgery
[2] Department of Pediatrics,Centre for Rare Diseases
[3] MBBM Foundation,Department for Women and Children’s Health, U.O.C. Inborn Metabolic Diseases
[4] ATS Monza e Brianza,Department of Pediatrics
[5] University of Milano-Bicocca,Department of Pediatrics
[6] University Hospital Santa Maria della Misericordia,Department of Translational Sciences, Pediatrics
[7] University Hospital,Department of Pediatrics, Meyer Children’s Hospital, Metabolic and Muscular Unit
[8] University of Bologna,Department of Clinical and Experimental Medicine, Metabolic Diseases, Pediatric Clinic
[9] University Magna Graecia,Department of Pathophysiology and Transplantation, Pediatric Highly Intensive Care Unit
[10] University Federico II,Department of Pediatrics
[11] Division of Metabolism Bambino Gesù Children’s Hospital,undefined
[12] University of Firenze,undefined
[13] University of Catania,undefined
[14] University of Milano,undefined
[15] IRCCS Ca’ Granda Ospedale Maggiore Policlinico Foundation,undefined
[16] University of Torino,undefined
[17] Rare Diseases Unit,undefined
[18] Pediatric Hospital Istituto Giannina Gaslini,undefined
来源：
Orphanet Journal of Rare Diseases | / 13卷
关键词：
Infantile onset Pompe disease; Alglucosidase alpha; ERT; Recombinant human GAA; rhGAA;
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