30 closest matches · ranked by relevance
Glucose Transporter Type 1 Deficiency Syndrome · GLUT1 Deficiency Syndrome
Primary: Number of Participants With Reduction in Spike-wave Fraction of the EEG Recording Time — 13 Participants
GLUT1DS1 · Epilepsy · Glut1 Deficiency Syndrome 1, Autosomal Recessive
Primary: Neuropsychological Score of Sustained Attention — 11 Participants
Glucose Transporter Type-1 Deficiency Syndrome (Glut1 DS)
Primary: Reported Change in Seizures Frequency From Baseline at 13 Weeks — 4.4; 189.5 seizures/two weeks
Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
Primary: Percent Reduction From Baseline to Week 8 in Frequency of Total Seizures (Normalized to a 4-Week Rate) — 12.6; 0.0 percent reduction of seizures per 4 wks — p=0.5812
Congenital Sucrase-isomaltase Deficiency (CSID)
Primary: Prevalence of CSID Genetic Variants — 27; 0 Participants
Diabetes Mellitus, Type 1
Primary: Glutathione Concentration — 541; 551 umol/L
Focal Segmental Glomerulosclerosis · Steroid Resistant Nephrotic Syndrome
Primary: Focal Segmental Glomerulosclerosis Permeability Factor (FSPF) — 0.69; 0.35 Palb — p=0.009
Glycogen Storage Disease Type IA · Von Gierke's Disease (GSD Type Ia)
Primary: Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs and Discontinuations Due to TEAEs — 3; 3; 3; 3 Participants
Pompe Disease · Glycogen Storage Disease Type II (GSD-II) · Acid Maltase Deficiency Disease
Primary: Recumbent Height/Length of Participants in Centimeters (cm) — 80.4; 93.8; 67.7; 91.1 cm
Adult Growth Hormone Deficiency · Hypothalamic-pituitary Disorders
Primary: Peak GH Level in Adult Patients With Hypothalamic-pituitary Disorders and 1-2 Pituitary Hormone Deficiency (PHD). — 0.9; 0.6; 0.7 ng/mL
Diabetes Mellitus, Type 1
Primary: Glutathione Concentration — 706; 734 umol/L
Diabetes Mellitus, Type 1
Primary: Change in Plasma Glucose — 81.4; 84.2; 54.0 mg/dL — p=<0.001
Type I Diabetes Mellitus
Primary: Insulin Sensitivity — 0.1; 0.07 mL/kg/min per uU/mL
Gaucher Disease, Type 1
Primary: Mean Change From Baseline to Month 9 in Hemoglobin (Hgb) Concentration for Each Treatment Group. — 1.624; 1.488 gram per deciliter (g/dl)
Pompe Disease
Primary: Safety Assessments of the rAAV1-CMV-GAA (Study Agent), Changes Post Study Agent Administration. — 40,031; 29,638; 5,509,882; 1,907,161 mU/mL
Gaucher Disease, Type 1
Primary: Change From Baseline to 12 Months in Hemoglobin Concentration for the 60 U/kg Treatment Group. — 2.429 g/dL — p=<0.0001
Gaucher Disease
Primary: Participants Who Experienced at Least One Adverse Event — 34 participants
Impaired Glucose Tolerance
Primary: Fasting Plasma Glucose — 77.4; 77.7 mmol/L
Diabetes Complications · Hypoglycemia
Primary: Percent Enrichment of Bicarbonate as Measured by Magnetic Resonance Spectroscopy (MRS) — 61.9 % enrichment — p=0.0053
Metabolic Disease · Seizures
Primary: Change From Baseline on the Adaptive Behavior Assessment System (ABAS) Test at the End of the Study Drug and Placebo Treatment Periods — 5.2; 4.5 scores on a scale
Type 1 Diabetes
Primary: Brain Glycogen Turnover Rate — 0.31; 0.28 mg/kg/min
Gaucher Disease, Type 1 · Cerebroside Lipidosis Syndrome · Glucocerebrosidase Deficiency Disease
Primary: Percentage of Participants Demonstrating A Meaningful Clinical Response — 77 percentage of participants
Duarte Galactosemia
Primary: Children's Memory Scale Score — 106.76; 104.15; 105.99; 103.78 score on a scale
Gaucher Disease, Type 3
Primary: Change From Baseline to 12 Months (Week 53) in Hemoglobin Concentration — 2.15 g/dL
GLUT1DS1
Primary: Change in Ketosis (Beta-hydroxybutyrate Levels) — 3.82 mM
Congenital Hyperinsulinism
Primary: Number of Subjects With Clinically Meaningful Reduction in Glucose Infusion Rate (Double-Blind) — 2; 0 Participants
Inflammatory Bowel Disease · Glycogen Storage Disease Type Ia
Primary: Serologic, Genetic and Inflammatory Markers Consistent With Inflammatory Bowel Disease — 11 participants testing positive
Diabetes Mellitus, Type 1
Primary: Maximum Epinephrine Response (LBGI Groups) — 495; 217 pg/ml
Gaucher Disease, Type 1
Primary: Number of Participants With Treatment-emergent Adverse Events Over Time — 6; 6; 6; 2 Participants
Schizophrenia · Psychotic Disorders
Primary: Brain Glycine Increments After Oral Glycine Administration Measured With MRS as Glycine/Total Creatine, Normalized to the Glycine Dose Administered (g/kg). — 393; 677…