Skip to content


The mission is to driven data to drive and advance research into the impact of nGD and improve patient health and social outcomes and lives.


Our vision is to create a global data resource, specific to neuronopathic Gaucher disease that will benefit patients and caregivers by increasing the understanding of the impact of the neurological manifestations of the disease, improve access to and achieve a timely diagnosis, to inform better care and support, to enable better targeted research, and the development of better safer treatments.


  • Owned and driven by the patient community
  • Independence
  • Transparency
  • Innovation
  • Respect
  • Quality
  • Integrity


To drive research that will advance disease management and treatment for nGD patients, through the development and maintenance of a global registry  


  • To report results of GARDIAN to all stakeholders, including clinical, scientific and GD2 and GD3 patient communities
  • To develop and validate disease-specific outcome measures for assessing HRQOL over the natural course of neuronopathic Gaucher disease
  • To improve our understanding of the self-reported burden over time, including changes in symptoms and HRQOL for patients with neuronopathic Gaucher disease, GD2 and GD3 globally
  • To improve our understanding of the demographic and disease-related characteristics of patients with neuronopathic Gaucher disease (GD2 and GD3) globally
  • To support development of standards of clinical management and quality of care
  • To facilitate clinical research through data sharing and provision of analytical services
  • To accelerate progress in understanding neuronopathic Gaucher disease, encouraging education of health care professionals and researchers
  • To support regulatory interactions between medicines agencies and pharmaceutical companies
  • To support network development and collaboration across the patient, clinical and research community
  • To protect patient privacy and ensure compliance with data protection of the highest standards 
  • To achieve patient guided clinical trials and better medicines