AVRO-RD-02-LTF01 A Long-Term Follow-up Study of Subjects With Gaucher Disease Who Previously Received AVR-RD-02 Summary: A follow-up study of subjects with Type I Gaucher Disease who previously received AVR-RD-02 gene therapy
G.R.I.T. STudyGetting global Rare disease Insights through Technology Summary: The G.R.I.T. Study is the first global app-based registered clinical trial for patients with metabolic disorders. It uses a mobile health journal, Zamplo, to record patient-reported...
GAUCHER, fabry, mps i, and pompe registriesSummary: Rare Disease Registries for Lysosomal Storage Disorders