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

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

Abstract 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 e...

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Bibliographic Details
Main Authors: Martin Magner, Zsuzsanna Almássy, Zoran Gucev, Beata Kieć-Wilk, Vasilica Plaiasu, Anna Tylki-Szymańska, Dimitrios Zafeiriou, Ioannis Zaganas, Christina Lampe
Format: Article
Language:English
Published: BMC 2022-05-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Morquio syndrome A
Mucopolysaccharidosis IVA
Central and South-Eastern European countries
Enzyme replacement therapy
Elosulfase alfa
Online Access:https://doi.org/10.1186/s13023-022-02332-7

Internet

https://doi.org/10.1186/s13023-022-02332-7

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