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
REN001Summary: A double-blind, placebo-controlled, study to evaluate the efficacy and safety of 24 weeks treatment with REN001 in patients with primary mitochondrial myopathy (PMM).
PTC923-MD-004-PKUSummary: A phase III study of PTC 923 in subjects with Phenylketonuria
CMVIBE TRIAL Summary: A phase 1/2A open-label dose-ranging and observer-blind placebo-controlled, safety and immunogenicity study of mRNA-1647 Cytomegalovirus vaccine in female and male participants 9 to 15 years of age and participants 16 to 25 years of age.
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...
The Canadian Fabry Disease Initiative Registry. Click here to learn more
GAUCHER, fabry, mps i, and pompe registriesSummary: Rare Disease Registries for Lysosomal Storage Disorders
MIGALASTAT SWITCHSummary: Evaluation of Biomarkers for patients receiving migalastat after switching from ERT.
Screening for Fabry cardiomyopathy using cardiac magnetic resonance imaging.