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Inborn Errors of Metabolism
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Inborn Errors of Metabolism trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
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Background: Turner Syndrome, galactosemia, and premature ovarian insufficiency are all conditions that may make it very hard or impossible for a person to beco…
This study is a Phase 3, non-randomized, multicenter, efficacy and safety study in adult patients with Gaucher disease Type 1, on stable treatment with enzyme r…
This is a Phase 1/2, open-label, multiple-center, dose escalation and cohort expansion study to evaluate the safety and efficacy of NGGT002 in adult subjects wi…
The Metabolism, Infection and Immunity (MINI) Study is a longitudinal natural history study at the National Institutes of Health (NIH) that aims to define the r…
Background: Niemann-Pick type C (NPC) disease is a rare, progressive neurodegenerative disease that affects mainly the brain, liver, and spleen but also other…
The purpose of this study is to evaluate the safety, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) of atumelnant treatment in pediatric participant…
Researchers plan to study the fat-rich particles, called lipoproteins, which circulate in the blood. This study is designed to improve understanding of normal,…
This is a Post Trial Access program for patients with Fabry Disease who previously participated in the ID-069A302 (MODIFY OLE) trial and who were still enrolled…
Long-term follow-up of subjects who received SB-318, SB-913, or SB-FIX in a previous trial and completed at least 52 weeks post-infusion follow-up in their prim…