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.

GARDIAN is patient-led and owned by the IGA as a global community to drive research and ensure that we work together with researchers and pharmaceutical companies to have one disease registry.

GARDIAN will be translated into 7 languages to start with, these will be UK English, US English, Arabic, Japanese, Chinese, German and French. 

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 for GARDIAN

We will strive to develop a registry that will capture clinical data from physicians/HCPs treating nGD patients and patient (caregiver)-reported data.

Our vision is that patients will be able to enrol in the registry as site patients (by their clinicians) and non-site patients (through our patient engagement portal) wherever you live in the world.

Using information, we collected from patients and caregivers and clinical experience with patients we have designed a nGD-specific patient-reported outcome (PRO) and caregiver-reported outcome (ObsRO). Through GARDIAN we want patients and caregivers to add their own data through a questionnaire that asks quality of life questions to capture the wider disease impact.