Harnessing the power of patients: Developing and validating the Neuronopathic Gaucher Disease Patient Reported Outcomes (nGD-PRO) and Observer Reported Outcomes (nGD-ObsRO) to measure HRQoL in patients with Gaucher Disease Type 2 and Type 3 documents over three and a half years of research to develop and assess the content validity of nGD-specific patient-reported outcomes (PRO) and observer-reported outcomes (ObsRO) measure in partnership with patients, caregivers, and other
stakeholders as part of the GARDIAN project.

GARDIAN is a patient-owned registry developed by the IGA together with Cerner Enviza and Aparito to improve Gaucher disease understanding, management and support for patients with Gaucher Disease Type 2 and Type 3. It also provides a research platform to provide evidence-based data for advancing disease management, designing safer treatments and improving patient outcomes.

This poster documents how the Neuronopathic Gaucher Disease Patient Reported Outcomes (nGD-PRO) and Observer Reported Outcomes (nGD-ObsRO) were developed and validated to measure HRQoL in patients with Gaucher Disease Type 2 and Type 3!

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