Medium-chain Acyl-coa Dehydrogenase Deficiency
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CLINICAL_TRIALS trial_result · 2026-04-21Clinical Trial Update: Study of Triheptanoin for the Prevention of Hypoglycemia in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD) [Phase 2]
A Phase 2 study of triheptanoin to prevent hypoglycemia in patients with Medium-chain acyl-CoA dehydrogenase deficiency has been suspended.
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CLINICAL_TRIALS trial_result · 2026-03-23Clinical Trial Update: High Intensity Exercise in Children With MCADD [NA]
A clinical trial evaluating high-intensity exercise in children with Medium-chain acyl-CoA dehydrogenase deficiency has completed. The snipp…
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CLINICAL_TRIALS research · 2026-03-10Clinical Trial Update: Metabolic Effects of Medium-Chain Fatty Acids in Patients With Medium-Chain Acyl-CoA Dehydrogenase Deficiency and Healthy Individuals [NA]
A clinical trial evaluating the metabolic effects of medium-chain fatty acids in patients with medium-chain acyl-CoA dehydrogenase deficienc…
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CLINICAL_TRIALS research · 2026-03-05Clinical Trial Update: Study to Evaluate the Use of Triheptanoin in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD) [Phase 2]
A Phase 2 clinical trial to evaluate triheptanoin in patients with Medium-chain Acyl-CoA Dehydrogenase Deficiency is currently recruiting.
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CLINICAL_TRIALS trial_result · 2025-09-26Clinical Trial Update: Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD) [Phase 2]
A Phase 2 clinical trial evaluating ACER-001 (sodium phenylbutyrate) for Medium-chain Acyl-CoA Dehydrogenase Deficiency has been suspended.
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CLINICAL_TRIALS research · 2025-07-11Clinical Trial Update: Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Pediatric and Adults Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD) [Phase 2]
A Phase 2 trial evaluating ACER-001 (sodium phenylbutyrate) for treatment of Medium-chain Acyl-CoA Dehydrogenase Deficiency in pediatric and…
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CLINICAL_TRIALS research · 2025-04-04Clinical Trial Update: Early Check: Expanded Screening in Newborns
An update on an ongoing clinical trial (NCT03655223) evaluating expanded newborn screening under the Early Check program, with enrollment by…
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CLINICAL_TRIALS trial_result · 2024-01-30Clinical Trial Update: Fatty Acid Oxidation Defects and Insulin Sensitivity [NA]
Clinical trial update reporting completed study on fatty acid oxidation defects and insulin sensitivity with posted results.
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CLINICAL_TRIALS trial_result · 2017-07-11Clinical Trial Update: Use of Ravicti™ in Patients With MCAD Deficiency With the 985A>G (K304E) Mutation [Phase 1]
Phase 1 trial update evaluating Ravicti in patients with MCAD deficiency carrying the 985A>G (K304E) mutation has completed with results pos…