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

BACKGROUND: Mucopolysaccharidosis IVA (MPS IVA), or Morquio A syndrome, is a rare inherited metabolic disorder caused by deficiency of the lysosomal enzyme N-acetylgalactosamine-6-sulfatase. A progressive systemic skeletal chondrodysplasia, leading to significant morbidity and reduced life expectanc...

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Detalles Bibliográficos
Autores principales:	Magner, Martin, Almássy, Zsuzsanna, Gucev, Zoran, Kieć-Wilk, Beata, Plaiasu, Vasilica, Tylki-Szymańska, Anna, Zafeiriou, Dimitrios, Zaganas, Ioannis, Lampe, Christina
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2022
Materias:	Position Statement
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9092811/ https://www.ncbi.nlm.nih.gov/pubmed/35538504 http://dx.doi.org/10.1186/s13023-022-02332-7

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