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Very Long-chain Acyl-CoA Dehydrogenase Deficiency
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Very Long-chain Acyl-CoA Dehydrogenase Deficiency trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which Very Long-chain Acyl-CoA Dehydrogenase Deficiency trials may be worth asking aboutNorth America
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The main goal of the project is provision of a global registry for mitochondrial disorders to harmonize previous national registries, enable world-wide particip…
Primary Mitochondrial diseases are a clinically and genetically heterogeneous group of disorders caused by mutations in genes encoded by nuclear Deoxyribonuclei…
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).
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approa…