30 closest matches · ranked by relevance
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 (MPS)
Primary: Number of Treatment Emergent Serious Adverse Events (SAE) — 5; 3; 2 events
Hurler Syndrome
Primary: Percentage of Adherence to the Scheduled Weekly Infusion by the Participants — 99 percentage
Sanfilippo Syndrome Type A (MPS IIIA)
Primary: Safety as Measured by Adverse Events Frequencies (by Type and Severity) — 174; 355; 0; 1 events
Mucopolysaccharidosis I
Primary: Number of Participants With Adverse Events — 3; 6; 7; 0 participants
Mucopolysaccharidosis I · Hurler Syndrome · Hurler-Scheie Syndrome
Primary: Safety Evaluation — 3; 0; 0; 1 participants
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
Mucopolysaccharidosis IVA · Morquio A Syndrome · MPS IVA
Primary: To Evaluate Safety and Tolerability of Infusions of BMN 110 at a Dose of 2.0 mg/kg/Week Over a 52-week Period in MPS IVA Subjects Less Than 5 Years of Age at Time of…
MPS IV A
Primary: Subject Incidence of Treatment Emergent AEs — 18; 18; 17; 17 participants
Mucopolysaccharidosis II
Primary: Ranked Adjusted 2-Component Composite Variable Score Based on Change From Baseline to Week 53 — 69.81; 50.86 sum of the ranked scores — p=0.0049
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 I · Hurlers Syndrome · Hurler-Scheie Syndrome
Primary: Overall Change From Baseline to Week 26 in Percent Predicted Forced Vital Capacity (FVC) — 54.2; 48.4; 51.5; 50.2 Percent predicted FVC
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 · 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
Mucopolysaccharidosis IIIB
Primary: Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs) — 1; 0; 1 Participants
Mucopolysaccharidosis I · Hurler Syndrome · Hurler-Scheie Syndrome
Primary: Safety Evaluation — 16; 4; 5; 3 participants
Mucopolysaccharidosis VI
Primary: 12-Minute Walk Test — 187; 118 meters
Mucopolysaccharidosis VI
Primary: Change From Baseline in 12-minute Walk Test at 24 Weeks — 109; 26 meters — p=0.025
Sanfilippo Syndrome Type A
Primary: Change From Baseline in Bayley Scales of Infant Development-III/Kaufman Assessment Battery for Children-II (BSID-III/KABC-II) Age-Equivalent Scores — 0.17; 1.19; -1.72…
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 Type 7
Primary: Percentage Change From Baseline in Urinary Glycosaminoglycan (uGAG) Dermatan Sulfate — -50.41; -38.94; -63.49; -51.82 percentage change
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
Mucopolysaccharidosis I · Hurler's Syndrome · Hurler-Scheie Syndrome
Primary: Change From Baseline to Week 182 in Percent Predicted Forced Vital Capacity (FVC) — 51.0; 48.4; 47.7; 47.2 percent predicted FVC
Hunter Syndrome
Primary: Change From Baseline in the Differential Ability Scales, Second Edition (DAS-II) General Conceptual Ability (GCA) Standard Score at Week 52 — -7.4; -4.4 Score on a scale…
Mucopolysaccharidosis I
Primary: Number of Participants Who Achieved Immune Tolerance Induction — 0; 1 participants
Hunter Syndrome
Primary: Height Overall — 113.759; 108.143 centimeter (cm) — p== 0.011
Mucopolysaccharidosis I · Mucopolysaccharidosis VI · Mannosidosis
Primary: Mean Percentage of Donor Cells in Study Population (Chimerism). — 85.8; 73.2; 84.6; 81.1 Percentage
Hunter Syndrome · Mucopolysaccharidosis II (MPS II)
Primary: Change From Baseline in Mean Percent Predicted Forced Vital Capacity (FVC) at Week 105 — -0.056 percent predicted FVC
Mucopolysaccharidoses
Primary: Carotid Intima-media Thickness, Measured in Millimeters — 0.44; 0.52; 0.56 mm
Hunter Syndrome
Primary: Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuation Due to TEAEs and Death — 5; 1; 1; 0 Participants