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

Martin Magner1, Zsuzsanna Almássy2, Zoran Gucev3

  • 1Department 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.

Orphanet Journal of Rare Diseases
|May 10, 2022
PubMed
Abstract

Keywords:
Central and South-Eastern European countriesElosulfase alfaEnzyme replacement therapyMorquio syndrome AMucopolysaccharidosis IVA

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