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VLCAD Deficiency
Clinical trial pipeline · Data from ClinicalTrials.gov
See which VLCAD Deficiency trials you may qualify forClinical trial pipeline · Data from ClinicalTrials.gov
See which VLCAD Deficiency trials you may qualify forPrimary Mitochondrial diseases are a clinically and genetically heterogeneous group of disorders caused by mutations in genes encoded by nuclear Deoxyribonuclei…
The main goal of the project is provision of a global registry for mitochondrial disorders to harmonize previous national registries, enable world-wide particip…
Ammonia is a waste product of protein and amino acid catabolism and is also a potent neurotoxin. High blood ammonia levels on the brain can manifest as cytotoxi…
Expanded access may be provided for qualified patients who have limited treatment options and are not eligible for a clinical trial.
The main goal of this study is to evaluate the effects of triheptanoin versus Medium-chain Triglycerides (MCT) on frequency of Major Clinical Events (MCEs).
The primary objective of this study is to assess the long-term safety, including pregnancy, infant, and lactation outcomes, of patients with LC-FAOD who are enr…