A global neuronopathic gaucher disease registry (GARDIAN): a patient-led initiative.
Tanya Collin-HistedMadeline StoodleyKathleen BeusterienDeborah ElsteinDena H JaffeShoshana Revel-VilkElin Haf Daviesnull nullPublished in: Orphanet journal of rare diseases (2023)
The comprehensive set of clinical and patient relevant outcomes data, developed collaboratively among all stakeholders, to be reported using GARDIAN will bridge the many gaps in the understanding of nGD and align with regulatory frameworks on real-world data needs.