FACTSSummary: Phase I gene therapy for Fabry Disease
FACTS
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sEARCH RESULTS
ST-920-201
ST-920-201Dose-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 DISEASE
GRIT STUDY
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...
CFDI Registry
The Canadian Fabry Disease Initiative Registry. Click here to learn more
GAUCHER, FABRY, MPS I, AND POMPE REGISTRIES
GAUCHER, fabry, mps i, and pompe registriesSummary: Rare Disease Registries for Lysosomal Storage Disorders
MIGALASTAT SWITCH
MIGALASTAT SWITCHSummary: Evaluation of Biomarkers for patients receiving migalastat after switching from ERT.
FABRY HCM
Screening for Fabry cardiomyopathy using cardiac magnetic resonance imaging.
PERIDOT – Patient ExpeRIence in fabry Disease On venglustaT
A randomized, double-blind, placebo-controlled, 12-month Phase III study to evaluate the effect of Venglustat on neuropathic and abdominal pain in male and female adults with Fabry disease who are treatment-naive or untreated for at least 6 months