1 active not recruiting, 1 completed. .
Follow Glycogen Storage Disease II: new trials by email Literature Review of Glycogen Storage Disease II trials Research Report on Glycogen Storage Disease II
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Early Check: Expanded Screening in Newborns Confirmatory Testing - Spinal Muscular Atrophy, Fragile X Syndrome | Active not recruiting | RTI International | 30000 | 2018-10-15 | 2026-07-01 | |
| Pompe Telemedicine Developmental Study Pompe Disease, Glycogen Storage Disease II | Completed | Duke University | 22 | 2015-12-01 | 2021-10-28 |
Glycogen Storage Disease Type II (2) · 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency (1) · 3-Hydroxyacyl-CoA Dehydrogenase Deficiency (1) · 3-methylcrotonyl CoA carboxylase 1 deficiency (1) · 3-methylcrotonyl CoA carboxylase 2 deficiency (1) · Acidemia, isovaleric (1) · Acrodermatitis enteropathica (1) · Adenine phosphoribosyltransferase deficiency (1) · Adrenal hyperplasia, congenital, type 5 (1) · Adrenocorticotropic hormone deficiency (1) · Amaurosis congenita of Leber, type 2 (1) · Anemia, Sickle Cell (1) · Angelman Syndrome (1) · Aortic Stenosis, Supravalvular (1) · Arginine-Glycine Amidinotransferase Deficiency (1)
Brain Diseases (2) · Brain Diseases, Metabolic (2) · Brain Diseases, Metabolic, Inborn (2) · Carbohydrate Metabolism, Inborn Errors (2) · Central Nervous System Diseases (2) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (2) · Genetic Diseases, Inborn (2) · Glycogen Storage Disease (2) · Lysosomal Storage Diseases (2) · Lysosomal Storage Diseases, Nervous System (2) · Metabolic Diseases (2) · Metabolism, Inborn Errors (2) · Nervous System Diseases (2) · Nutritional and Metabolic Diseases (2) · Abnormalities, Multiple (1)
Duke University (1) · RTI International (1)
United States (2)
Curt Scharfe, MD, PhD (1) · Priya Kishnani (1)