Liver Glycogen Storage Disease Top 10

  1. What are the best options (for example gene therapy or enzyme replacement therapy) for achieving sufficient amount of working enzyme in patients with liver GSD?
  2. Can consensus guidelines (for management) be achieved for patients with liver GSD?
  3. How should optimal metabolic control both clinically and biochemically (like lactate, ketones and/or lipids) be achieved in liver GSD?
  4. How should sickness and emergency situations be managed for patients with liver GSD?
  5. What is the best way to start dietary treatment, finding the optimal doses, and to administer the diet for patients with liver GSD?
  6. How can existing cornstarch preparations be modified or alternative treatments be implemented that are easier to administer and/or keep blood sugar levels more stable for patients with liver GSD?
  7. What is the role for new methods for monitoring metabolic control (like noninvasive continuous glucose and lactate measurements, new biomarkers) for patients with liver GSD?
  8. How to manage diet regimen in relation to "before, during and after" physical exercise (sport, playing) for patients with liver GSD?
  9. What are the long-term complications (liver, renal, gut) of a diet rich in uncooked cornstarch and/or high protein and should the diet be adjusted to prevent complications in liver GSD?
  10. What are the risks and benefits of different options for overnight treatment for patients with liver GSD and how can we maximize safety?
  11. How to prevent and/or treat muscle problems in patients with liver GSD?