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
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.
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.