Cardiac involvement in MPS patients: incidence and response to therapy in an Italian multicentre study.
Simona SestitoGiada RinninellaAngelica RampazzoFrancesca D'AvanzoLucia ZampiniLucia SantoroOrazio GabrielliAgata FiumaraRita BaroneNicola VolpiMaurizio ScarpaRosella TomaninDaniela ConcolinoPublished in: Orphanet journal of rare diseases (2022)
In conclusion, our analysis confirms the wide presence of cardiopathies, at different extent, in the MPS population. Since cardiac pathology is the main cause of death in many MPS subtypes, it is necessary to raise awareness among cardiologists about early cardiac morpho-structural abnormalities. The encouraging data regarding the long-term effects of ERT, also on heart damage, underlines the importance of an early diagnosis and timely start of ERT.