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Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program ClinicalTrials.gov ID NCT04632953 Sponsor Ultragenyx Pharmaceutical Inc Information provided by Ultragenyx Pharmaceutical Inc (Responsible Party) Last Update Posted 2024-08-29 Study Overview The primary objective of this...

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...