Records containing: Metabolism, Inborn Errors
This is the wording recorded or indexed by the source. An agency index term is not necessarily an authorised indication. Co-listed drug names are not linked to this term as a treatment relationship.
Follow recorded terms in Drug Alerts for Android. Free includes unlimited local condition follows and an on-open recent-record watchlist. Plus adds no ads and personal organization tools.
All
FDA submission records
EMA medicine records
ClinicalTrials.gov record updates
ClinicalTrials.gov records with results posted
-
CLINICAL_TRIALS study record update · 2026-04-16MAGNITUDE-2: A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of NTLA-2001 in Participants With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN)
-
CLINICAL_TRIALS study record update · 2025-04-06Safety and Efficacy of Treatment With Large Neutral Amino Acids in Patients With Classical Phenylketonuria
-
CLINICAL_TRIALS study record update · 2024-12-04Cardiac Manifestations in Adult Patients With Inherited Metabolic Disease: a Case Series
-
CLINICAL_TRIALS study record update · 2023-02-13Biomarker for Hurler Disease - An International, Multicenter, Epidemiological Protocol
-
CLINICAL_TRIALS study record update · 2022-11-02Evaluation of Genetic Etiology in Patients With Cerebral Palsy
- Organize your saved records without ads with Plus.
-
CLINICAL_TRIALS study record update · 2022-03-02Prospective Study on the Impact of COVID-19 on the Equilibrium of Hereditary Metabolic Disease (MHM) in Patients With or Who Have Contracted COVID-19
-
CLINICAL_TRIALS study record update · 2021-03-17Natural History Study of Children With Metachromatic Leukodystrophy
-
CLINICAL_TRIALS study record update · 2020-11-13A Global, Phase 1/2, Open Label, Dose Escalation Study to Evaluate the Safety, Pharmacodynamics, and Pharmacokinetics of mRNA-3704 in Patients With Isolated Methylmalonic Acidemia Due to Methylmalonyl-CoA Mutase Deficiency
-
CLINICAL_TRIALS study record update · 2020-07-08North Carolina Newborn Exome Sequencing for Universal Screening
-
CLINICAL_TRIALS study record update · 2019-10-16Fat and Sugar Metabolism During Exercise in Patients With Metabolic Myopathy
-
CLINICAL_TRIALS study record update · 2015-12-21An Open-label Extension Study to Assess the Long-term Safety and Efficacy of ISIS 301012 in Patients With Familial Hypercholesterolemia or Severe-Hypercholesterolemia
-
CLINICAL_TRIALS study record update · 2015-06-24A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Effect of Apolipoprotein B(ApoB) Reduction by ISIS 301012 on Liver Triglyceride Content in Subjects With Varying Degrees of Hyperlipidemia
-
CLINICAL_TRIALS study record update · 2014-12-09ClinicalTrials.gov study NCT00010361
-
CLINICAL_TRIALS study record update · 2013-04-05An Open-Label Extension Study to Assess the Long-term Safety and Efficacy of Mipomersen in Subjects With Familial Hypercholesterolemia
-
CLINICAL_TRIALS study record update · 2013-03-21A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety and Efficacy of Mipomersen as Add-on Therapy in Homozygous Familial Hypercholesterolemia Subjects
-
CLINICAL_TRIALS study record update · 2008-03-04A Study to Assess Adding Ezetimibe 30 mg to Ongoing Treatment With Ezetimibe 10 mg in Patients With Homozygous Sitosterolemia