Cardiac involvement in MPS patients: incidence and response to therapy in an Italian multicentre study

Cardiac involvement in MPS patients: incidence and response to therapy in an Italian multicentre study

Abstract Background Mucopolysaccharidoses (MPSs) are a group of lysosomal storage disorders caused by the deficit of lysosomal hydrolases involved in the degradation of glycosaminoglycans (GAGs). The course is chronic and progressive, with multisystemic involvement that often leads to cardiovascular...

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Bibliographic Details
Main Authors: Simona Sestito, Giada Rinninella, Angelica Rampazzo, Francesca D’Avanzo, Lucia Zampini, Lucia Santoro, Orazio Gabrielli, Agata Fiumara, Rita Barone, Nicola Volpi, Maurizio Scarpa, Rosella Tomanin, Daniela Concolino
Format: Article
Language:English
Published: BMC 2022-06-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Mucopolysaccharidoses (MPS)
Glycosaminoglycans (GAGs)
Heart involvement
Valvulopathies
Enzyme replacement therapy (ERT)
Online Access:https://doi.org/10.1186/s13023-022-02396-5

Internet

https://doi.org/10.1186/s13023-022-02396-5

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