Publikace na 1. lékařská fakulta |
2014
Abstrakt
Thus far, there have been only a few publications addressing the problem of enzyme replacement therapy (ERT) cessation in mucopolysaccharidoses. In our previous study of MPS II, rapid cessation of ERT resulted not only in loss of the beneficial effects of treatment, but also in a significant worsening of patients' clinical status.
Our current study describes the effects of ERT interruption in additional patients with MPS II (n = 1) and VI (n = 4) from Poland, Lithuania and Czech Republic.