Clinical outcomes in idursulfase-treated patients with mucopolysaccharidosis type II: 3-year data from the hunter outcome survey (HOS).

Joseph MuenzerRoberto GiuglianiMaurizio ScarpaAnna Tylki-SzymańskaVirginie JegoMichael Beck

Published in: Orphanet journal of rare diseases (2017)

These findings suggest that idursulfase has a positive effect on uGAG levels, 6MWT results, LVMI, FVC, FEV1 and hepatosplenomegaly after 1, 2 and 3 years treatment.

Keyphrases

replacement therapy
electronic health record
cross sectional
combination therapy
newly diagnosed
artificial intelligence