by Elleine Allapitan | Jul 5, 2023
FACTS Summary: Phase I gene therapy for Fabry...
by Elleine Allapitan | Jul 5, 2023
ST-920-201 Dose-ranging study of ST-290, an AAV2/6 Human Alpha Galactosidase – a gene therapy in subjects with Fabry disease DOSE-RANGING STUDY OF ST-920, AN AAV2/6 HUMAN ALPHA GALACTOSIDASE A GENE THERAPY IN SUBJECTS WITH FABRY...
by Elleine Allapitan | Jul 5, 2023
G.R.I.T. STudy Getting 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...
by Elleine Allapitan | Jul 5, 2023
CFDI Canadian Fabry Disease Initiative...
by Elleine Allapitan | Jul 4, 2023
GAUCHER, fabry, mps i, and pompe registries Summary: Rare Disease Registries for Lysosomal Storage...
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