GSDII or Pompe Disease

GSDII (for Glycogen Storage Disease type II), or Pompe Disease, is part of a group of metabolic genetic diseases, glycogen diseases, for which the organism is unable to correctly use sugar as a power source due to an enzyme deficiency.


Immune Tolerance Induction Study — Myozyme

  • Reference laboratory: Institute of Myology

This exploratory, open-labeled study of patients with Pompe disease, who have previously received Myozyme (alglucosidase alfa) treatment aims at evaluating the efficacy, safety and clinical benefit of 2 Immune Tolerance Induction (ITI) regimens in combination with Myozyme.

For more information: the immune tolerance induction study webpage

NEO-EXT study — LTS 13769

  • Reference laboratory: Institute of Myology

An open-label, multicenter, multinational extension study of the long-term safety and pharmacokinetics of repeated biweekly infusions of NeoGAA in patients with Pompe Disease.

For more information: NEO-EXT study webpage