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Consensus statement on enzyme replacement therapy for mucopolysaccharidosis IVA in Central and South-Eastern European countries.

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Title: Consensus statement on enzyme replacement therapy for mucopolysaccharidosis IVA in Central and South-Eastern European countries.
Authors: Magner M; Department of Paediatrics and Inherited Metabolic Disorders, General University Hospital and First Faculty of Medicine, Charles University, KPDPM 1. LF UK a VFN v Praze, Ke Karlovu 2, 128 08, Prague, Czech Republic. martin.magner@vfn.cz., Almássy Z; Department of Toxicology and Metabolic Diseases, Heim Pal National Pediatric Institute, Budapest, Hungary., Gucev Z; University Children's Hospital, Skopje, North Macedonia., Kieć-Wilk B; Unit of Rare Metabolic Diseases, Department of Metabolic Diseases, Jagiellonian University Medical College, University Hospital, Krakow, Poland., Plaiasu V; Regional Centre of Medical Genetics, INSMC Alessandrescu-Rusescu, Bucharest, Romania., Tylki-Szymańska A; Department of Pediatric Nutrition and Metabolic Diseases, The Children's Memorial Health Institute, Warsaw, Poland., Zafeiriou D; First Department of Pediatrics, Hippokratio General Hospital, Aristotle University, Thessaloniki, Greece., Zaganas I; Neurogenetics Laboratory, Neurology Department, University Hospital of Heraklion, University of Crete, Heraklion, Greece., Lampe C; Department of Child Neurology, Epileptology and Social Pediatrics, Centre for Rare Diseases, University of Giessen, Giessen, Germany.
Source: Orphanet journal of rare diseases [Orphanet J Rare Dis] 2022 May 10; Vol. 17 (1), pp. 190. Date of Electronic Publication: 2022 May 10.
Publication Type: Journal Article; Research Support, Non-U.S. Gov't; Consensus Statement
Journal Info: Publisher: BioMed Central Country of Publication: England NLM ID: 101266602 Publication Model: Electronic Cited Medium: Internet ISSN: 1750-1172 (Electronic) Linking ISSN: 17501172 NLM ISO Abbreviation: Orphanet J Rare Dis Subsets: MEDLINE
Database: MEDLINE Ultimate
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ISSN:1750-1172
DOI:10.1186/s13023-022-02332-7