A Study of Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidosis, or Gaucher Disease Type 2 in Pediatric Patients
Rochester, MN
The objectives of this study are to collect data on survival of pediatric patients with early onset of either GM1 gangliosidosis, GM2 gangliosidoses, or GD2, to collect data that can constitute a historical control group for future clinical trials, and to evaluate whether the 3 conditions can be assessed together in a single clinical trial.