metabolism, inborn errors
Showing 1 - 15 of 15
Cerebral Palsy, Genetic Disease, Developmental Delay Trial in Ljubljana (NGS)
Completed
- Cerebral Palsy
- +3 more
- NGS
-
Ljubljana, SloveniaUniversity Children's Hospital, University Medical Centre Ljublj
Nov 1, 2022
COVID-19 and Hereditary Metabolic Diseases
Recruiting
- Covid19
- Metabolism, Inborn Errors
-
Lille, FranceHop Claude Huriez Chu Lille
Feb 28, 2022
Cardiac Manifestation and Inherited Metabolic Diseases
Recruiting
- Metabolism, Inborn Errors
-
Lausanne, Vaud, SwitzerlandLausanne University Hospital
Aug 7, 2021
Biomarker for Hurler Disease (BioHurler)
Active, not recruiting
- Mucopolysaccharidosis Type I
- +2 more
-
Cairo, Egypt
- +4 more
May 12, 2021
Children With Metachromatic Leukodystrophy
Terminated
- Lipid Metabolism Disorders
- +13 more
-
Torrance, California
- +13 more
Mar 16, 2021
Methylmalonic Acidemia (MMA), Metabolism, Inborn Errors Trial (mRNA-3704)
Withdrawn
- Methylmalonic Acidemia (MMA)
- Metabolism, Inborn Errors
- mRNA-3704
- (no location specified)
Nov 10, 2020
Metabolism, Inborn Errors, Lipid Metabolism, Inborn Errors, Carbohydrate Metabolism, Inborn Errors Trial in Copenhagen (Sugar)
Unknown status
- Metabolism, Inborn Errors
- +22 more
- Sugar
-
Copenhagen, DenmarkNeuromuscular Research Unit, 3342
Oct 15, 2019
Lipid Metabolism, Inborn Errors, Hyperlipidemias, Metabolic Diseases Trial in Amsterdam (mipomersen, Placebo)
Completed
- Lipid Metabolism, Inborn Errors
- +13 more
- mipomersen
- Placebo
-
Amsterdam, Netherlands(unnamed)
Sep 9, 2016
Lipid Metabolism, Inborn Errors, Hypercholesterolemia, Autosomal Dominant, Hyperlipidemias Trial in United States (mipomersen
Completed
- Lipid Metabolism, Inborn Errors
- +13 more
- mipomersen sodium
-
Chicago, Illinois
- +4 more
Aug 1, 2016
Lipid Metabolism, Inborn Errors, Hypercholesterolemia, Autosomal Dominant, Hyperlipidemias Trial in Worldwide (Mipomersen
Completed
- Lipid Metabolism, Inborn Errors
- +13 more
- Mipomersen Sodium
-
Mission Viejo, California
- +32 more
Aug 1, 2016
Lipid Metabolism, Inborn Errors, Hypercholesterolemia, Autosomal Dominant, Hyperlipidemias Trial in Worldwide (mipomersen,
Completed
- Lipid Metabolism, Inborn Errors
- +13 more
- mipomersen
- Placebo
-
Charlotte, North Carolina
- +9 more
Aug 1, 2016
Metabolism, Inborn Errors, Granulomatous Disease, Chronic Trial in Seattle (cyclosporine, fludarabine, mycophenolate mofetil)
Completed
- Metabolism, Inborn Errors
- Granulomatous Disease, Chronic
- cyclosporine
- +2 more
-
Seattle, WashingtonFred Hutchinson Cancer Research Center
Dec 8, 2014
Methionine Adenosyltransferase Deficiency, Metabolism, Inborn Errors Trial (phosphatidylcholine)
Completed
- Methionine Adenosyltransferase Deficiency
- Metabolism, Inborn Errors
- (no location specified)
Jun 23, 2005
Diagnostic and Screening Study of Genetic Disorders
Completed
- Tay-Sachs Disease
- +3 more
-
New York, New YorkMount Sinai School of Medicine
Jun 23, 2005