30 closest matches · ranked by relevance
Lysosomal Acid Lipase Deficiency
Primary: Percentage Of Participants Achieving Alanine Aminotransferase Normalization — 31; 7; 56; 37 percentage of participants — p=0.0271
Lysosomal Acid Lipase Deficiency
Primary: Participants Experiencing Severe Treatment-emergent Adverse Events (TEAEs) — 1; 1; 2 Participants
Cholesterol Ester Storage Disease(CESD) · Lysosomal Acid Lipase Deficiency · LAL-Deficiency
Primary: Number Of Participants Reporting TEAEs And Infusion-Related Reactions (IRRs) — 1; 3; 3; 0 participants
Lysosomal Acid Lipase Deficiency · Wolman Disease
Primary: Percentage Of Participants In The Primary Efficacy Analysis Set (PES) Surviving To 12 Months Of Age — 67 Percentage of participants
Cholesterol Ester Storage Disease (CESD) · Lysosomal Acid Lipase Deficiency · LAL-Deficiency
Primary: Number Of Participants Reporting TEAEs And IARs — 8; 1; 2; 0 Participants
Sphingomyelin Lipidosis
Primary: Number of Participants With Treatment Emergent Adverse Events (TEAEs), Treatment Emergent Serious Adverse Events (TESAEs) and Adverse Events of Special Interest (AESIs)…
Sphingomyelin Lipidosis
Primary: Percent Predicted (% Predicted) Hemoglobin (Hb) and Altitude-Adjusted Diffusing Capacity of the Lung for Carbon Monoxide (DLco) at Baseline — 48.45; 49.44 % Predicted DLco
Hepatic Steatosis
Primary: Hepatic Triglyceride (%) — 15.9; 14.8 Fat/Fat+Water (%) — p=0.42
Familial Partial Lipodystrophy
Primary: Change in the Liver Triglycerides (TG). — 13.2; 6.4; 12.3; 10.6 percentage of liver triglycerides
Hyperlipoproteinemia Type I · Hypertriglyceridemia
Primary: Fasting Serum Triglycerides — 705; 1827; 1511; 1007 mg/dL
Exocrine Pancreatic Insufficiency · Cystic Fibrosis
Primary: Treatment Difference in Coefficient of Fat Absorption (CFA) Change From Baseline — -11.852 percent change from baseline
Sphingomyelin Lipidosis
Primary: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) — 4; 9; 7; 20 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
Pompe Disease (Late-Onset) · Glycogen Storage Disease Type II (GSD-II) · Glycogenesis Type II
Primary: Summary of Participants Reporting Treatment-Emergent Adverse Events For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600) — 60; 37; 21…
Smith-Lemli-Opitz Syndrome
Primary: Change in Plasma Cholesterol — 37.8 change (%) compared to baseline — p=0.011
Familial Partial Lipodystrophy
Primary: Percent Change From Baseline in Fasting Triglycerides Levels at End of the Treatment (Week 27) — -59.9 percent change
Lipodystrophy
Primary: Change in Log 10 Fasting Triglycerides. — 0.1; -0.2 log 10 mg/dl
Acid Sphingomyelinase Deficiency
Primary: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) — 3; 0 Participants
Refractory Angina · Raised Lipoprotein(a)>50mg/dL or >500mg/L
Primary: Changes in Quantitative Myocardial Perfusion Measured by Stress/Rest Cardiovascular Magnetic Resonance Imaging — 0.47; -0.16 ratio — p=0.001
Sanfilippo Syndrome Type A (MPS IIIA)
Primary: Safety as Measured by Adverse Events Frequencies (by Type and Severity) — 174; 355; 0; 1 events
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
Bile Acid Synthesis Defect · Inborn Error of Bile Acid Metabolism · Inborn Error of Bile Acid Conjugation
Primary: Conjugated Cholic Acid (GCA) for the Treatment of Inborn Errors in Bile Acid Synthesis Involving Side-chain Conjugation. — 5 Participants
Familial Partial Lipodystrophy · Hypertriglyceridemia · Fatty Liver
Primary: Change in Fasting Serum Triglyceride (at 12 Weeks) — -0.44; -20.27 percent change — p=0.517
Niemann-Pick Disease, Type C
Primary: Efficacy of Adrabetadex (VTS-270) to Reduce Plasma Levels of a Conjugated Bile Acid, Known as 5α-cholanic Acid-3β, 5α, 6β-triol N-(Carboxymethyl)-Amide — 32.6; 87.9 ng/ml
GM2 Gangliosidosis · Tay-Sachs Disease · Sandhoff Disease
Primary: Clinical Impression of Change in Severity (CI-CS) [Fields et al 2021] — 0.71 score on a scale — p=0.044
Pompe Disease · Glycogen Storage Disease Type II (GSD-II) · Glycogenesis 2 Acid Maltase Deficiency
Primary: Participants' Efficacy Response During the Treatment Period as Compared to Baseline for Participants With Respiratory Decline on Standard Treatment — 0; 0; 0; 1…
Exocrine Pancreatic Insufficiency · Cystic Fibrosis
Primary: Safety — 3; 0 Participants
Familial Chylomicronemia Syndrome (FCS)
Primary: Percent Change in Fasting Triglycerides From Baseline to 12 Weeks — 45.6; 3.7; -13.9 percent change — p=0.0538
Sanfilippo Syndrome Type A (MPS IIIA)
Primary: Safety as Measured by Adverse Events Frequencies (by Type and Severity) — 53; 128; 0; 1 number of events
Pompe Disease (Late-onset) · Glycogen Storage Disease Type II (GSD-II) · Acid Maltase Deficiency Disease
Primary: Summary of Patients Reporting Treatment-Emergent Adverse Events — 60; 30; 32; 17 participants