3,998 trials (keyword fallback)
Mucopolysaccharidosis IIIB
Primary: Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs) — 1; 0; 1 Participants
Sanfilippo Syndrome
Primary: Number of Participants With Overall Response Using Bayley Scales of Infant Development Assessment Third Edition (BSID-III) — 0; 2; 1 participants — p=0.4615
Mucopolysaccharidosis Type I · Mucopolysaccharidosis Type II · Mucopolysaccharidosis Type VI
Primary: Children's Health Questionnaire - Parent Form 50 Bodily Pain Standardized Score — 1.4; 1.0 units on a scale
Mucopolysaccharidosis I
Primary: Number of Participants With Adverse Events — 3; 6; 7; 0 participants
Mucopolysaccharidosis I · Hurler's Syndrome · Hurler-Scheie Syndrome
Primary: Percent Change From Baseline to Week 26 in Urinary Glycosaminoglycan (GAG) Level — -57.57; -66.63; -66.15; -62.66 Percentage of Change in GAG Level
MPS IV A
Primary: Change From Baseline in Endurance as Measured by the 6-minute Walk Test — 211.9; 205.7; 203.9; 225.4 meters — p=0.0174
Mucopolysaccharidosis IV A · Morquio A Syndrome · MPS IVA
Primary: Change From Baseline in 6-minute Walk (6MW) Test - ITT — 219.7; 207.2; 205.7; 203.9 meters
Mucopolysaccharidosis VI
Primary: 12-Minute Walk Test — 187; 118 meters
MPS IV A
Primary: Subject Incidence of Treatment Emergent AEs — 18; 18; 17; 17 participants
Sly Syndrome · MPS VII · Mucopolysaccharidosis
Primary: Percent Change From Baseline in uGAG Excretion (LC-MS/MS-DS) at Week 48 — -58.17 percent change — p=< 0.0001
Sly Syndrome · MPS VII · Mucopolysaccharidosis
Primary: Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation — 12; 4; 9; 1 Participants
Mucopolysaccharidosis Type 7
Primary: Percentage Change From Baseline in Urinary Glycosaminoglycan (uGAG) Dermatan Sulfate — -50.41; -38.94; -63.49; -51.82 percentage change
MPS 7 · Sly Syndrome · Mucopolysaccharidosis
Primary: European Union (EU) and Rest of World: Percentage Change From Baseline in Urinary Glycosaminoglycan (uGAG) Dermatan Sulfate (DS) at UX003 Treatment Week 24 — -64.82…
Type 1 Diabetes Mellitus · Type 2 Diabetes Mellitus · Hypercholesterolemia
Primary: Changes in LDL Cholesterol — -0.83; -1.47; -0.93; -0.32 mmol/L
Diabetes Mellitus, Type 1
Primary: Mean Stimulated C-peptide Area Under the Curve — 0.28; 0.27; 0.25 pmol/ml — p=0.47