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Sanofi Genzyme Rare Disease Registry - A Multi-Center, International, Longitudinal, Observational program designed to track natural history and outcomes of patients with Gaucher, Fabry, MPSI, and Pompe Disease ( Gaucher registry, Fabry registry, MPSI registry, Pompe registry) International...

A Phase 1/2 Open-Label, Dose Escalation Study to Determine the Safety and Efficacy of BMN 307, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Phenylalanine Hydroxylase in Subjects with Phenylketonuria and Plasma Phe Levels > 600 μmol/L ClinicalTrials.gov ID NCT04480567 Sponsor...

Thromboxane A2 blockade attenuates ethanol-induced myocardial inflammation: Sipping from the same bottle. Originally, light-to-moderate alcohol consumption was believed to provide some protective effects on the cardiovascular system through its antioxidant properties and other constituents; this was...