Our Phase II Study – RAINBOW RAndomized, double-blINd, placeBo-controlled, multicenter, 12-week study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of Oral AZ-3102 in patients With GM2 gangliosidosis or Niemann-Pick type C (NPC) diseases. This study aims to assess the correct doses of AZ-3102 to treat GM2 and NPC diseases by evaluating the clearance of […]

PRONTO study

Our Natural History study Prospective longitudinal study of neurological disease trajectory in children living with late-infantile or juvenile onset of GM1 or GM2 gangliosidoses, PRONTO. To gain more insight in the natural development of the disease, we designed PRONTO (PROspective Neurological Disease TrajectOry Study), a prospective Natural History study. By enrolling children with late infantile […]