The landscape of Mucopolysaccharidosis in Southern and Eastern European countries: a survey from 19 specialistic centers

Abstract Background Mucopolysaccharidoses (MPS) are a group of lysosomal storage disorders caused by defects in genes coding for different lysosomal enzymes which degrade glycosaminoglycans. Impaired lysosomal degradation causes cell dysfunction leading to progressive multiorgan involvement, disabli...

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Autores principales:	Anna Tylki-Szymańska, Zsuzsanna Almássy, Violetta Christophidou-Anastasiadou, Daniela Avdjieva-Tzavella, Ingeborg Barisic, Rimante Cerkauskiene, Goran Cuturilo, Maja Djiordjevic, Zoran Gucev, Anna Hlavata, Beata Kieć-Wilk, Martin Magner, Ivan Pecin, Vasilica Plaiasu, Mira Samardzic, Dimitrios Zafeiriou, Ioannis Zaganas, Christina Lampe
Formato:	Artigo
Lenguaje:	Inglês
Publicado:	BMC 2022-03-01
Colección:	Orphanet Journal of Rare Diseases
Materias:	Mucopolysaccharidoses Morquio A syndrome Southern and Eastern European countries Enzyme replacement therapy Treatment accessibility
Acceso en línea:	https://doi.org/10.1186/s13023-022-02285-x
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