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Record #49661

An Innovative Tool for Evidence-Based, Personalized Treatment Trials in Mucopolysaccharidosis

Publisher
MDPI
Original paper date
May 22, 2023
Institution
Institute of Congenital Metabolic Diseases, Paracelsus Medical University, 5020 Salzburg, Austria · European Reference Network for Hereditary Metabolic Diseases, MetabERN, 33100 Udine, Italy · Stem Cell and Neurotherapies, Division of Cell Matrix Biology and Regenerative Medicine, Faculty of Biology, Medicine and Health, University of Manchester, Manchester M13 9PL, UK · Department of Genetics, Medical Genetics Service and Biodiscovery Laboratory, Portal Hospital de Clínicas de Porto Alegre, Universidade Federal do Rio Grande do Sul (UFRGS), Casa dos Raros, Porto Alegre 90610-261, Brazil · Department of Child Neurology, Epilepetology and Social Pediatrics, Center of Rare Diseases, University Hospital Giessen/Marburg, 35392 Giessen, Germany · Regional Coordinating Center for Rare Diseases, University Hospital Udine, 33100 Udine, Italy · Department of Neurology, Christian Doppler University Hospital, Paracelsus Medical University, 5020 Salzburg, Austria · Department of Pediatric Cardiology, University Hospital Mainz, 55131 Mainz, Germany · Team Biostatistics and Big Medical Data, IDA Lab Salzburg, Paracelsus Medical University, 5020 Salzburg, Austria · Research and Innovation Management, Paracelsus Medical University, 5020 Salzburg, Austria
Open access
No
Article type
Research Article
Broad categories
(B/T) Data Science(BLS) Biochemistry(BLS) Genetics(HSC) Medicine - Endocrinology
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100.0%
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