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Assessing the Role of Anti rh-GAA in Modulating Response to ERT in a Late-Onset Pompe Disease Cohort from the Italian GSDII Study Group.

Massimiliano MirabellaStefano Cotti PiccinelliSabrina RavagliaSerenella ServideiMaurizio MoggioOlimpia MusumeciMaria Alice DonatiElena PegoraroAntonio Di MuzioLorenzo MaggiPaola ToninGianni MarrosuCristina SancriccaAlberto LerarioMichele SacchiniClaudio SempliciniVirginia BozzoniRoberta TeleseSilvia BonannoRachele PirasMaria Antonietta MaioliGiulia RicciLiliana VercelliAnna GalvagniSerena Gallo CassarinoFilomena CariaTiziana MonginiGabriele SicilianoAlessandro PadovaniAntonio Toscano
Published in: Advances in therapy (2019)
Our results confirm that in a large cohort of LOPD patients, anti rh-GAA antibody generation did not significantly affect either clinical outcome or ERT efficacy. However, in the first 36 months of treatment, a possible interference of low-medium antibody titers with the clinical status could be present. Therefore, a careful and regular evaluation of antibody titers, especially in cases with evidence of clinical decline despite ERT, should be performed.
Keyphrases
  • late onset
  • early onset
  • end stage renal disease
  • newly diagnosed
  • ejection fraction
  • chronic kidney disease
  • peritoneal dialysis
  • prognostic factors
  • replacement therapy
  • signaling pathway
  • patient reported outcomes