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VLCAD Deficiency
Clinical trial pipeline · Data from ClinicalTrials.gov
See which VLCAD Deficiency trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
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Primary 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…
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…
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).