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The Neuronopathic Gaucher Registry
Owned and governed by the global patient community

Welcome to the GARDIAN website, the Gaucher Registry for Development, Innovation & Analysis of Neuronopathic disease.

GARDIAN is a global registry and open to all nGD patients and their caregivers worldwide

First phase of recruitment closing in

Register now!

Register for GARDIAN

What is GARDIAN?

  • A registry to improve disease understanding, management and support for patients with GD2 and GD3. Designed to study patients with GD2 and GD3 worldwide in a systematic and standardized manner
  • A research platform to provide evidence-based data for advancing disease management, designing safer treatments and improving patient outcomes.

What will it seek to do?

  • Characterize patient populations with GD2 and GD3.
  • Study the natural history of GD2 and GD3.
  • Evaluate psychometric properties of nGD-specific patient-reported outcome (PRO) and caregiver-reported outcome (ObsRO). 
  • Examine current clinical care guidelines and definitions
    • Understand adherence to current nGD guidelines to achieve international harmonization
    • Examine severity scoring
    • Examine diagnostic criteria
  • Safety monitoring
  • Identify patient populations eligible for clinical trials or other research projects

Our vision is a world where all Gaucher patients have access to the treatment and care they need and there is the possibility of a cure.