30 closest matches · ranked by relevance
Sanfilippo Syndrome Type A (MPS IIIA)
Primary: Safety as Measured by Adverse Events Frequencies (by Type and Severity) — 174; 355; 0; 1 events
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
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 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…
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 (MPS)
Primary: Number of Treatment Emergent Serious Adverse Events (SAE) — 5; 3; 2 events
MPS IV A
Primary: Subject Incidence of Treatment Emergent AEs — 18; 18; 17; 17 participants
Hurler Syndrome
Primary: Percentage of Adherence to the Scheduled Weekly Infusion by the Participants — 99 percentage
Mucopolysaccharidosis IIIB
Primary: Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs) — 1; 0; 1 Participants
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 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
Mucopolysaccharidosis VI
Primary: Change From Baseline in 12-minute Walk Test at 24 Weeks — 109; 26 meters — p=0.025
Mucopolysaccharidosis VI
Primary: 12-Minute Walk Test — 187; 118 meters
Mucopolysaccharidosis I · Hurler Syndrome · Hurler-Scheie Syndrome
Primary: Safety Evaluation — 3; 0; 0; 1 participants
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
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
Mucopolysaccharidosis Type 7
Primary: Percentage Change From Baseline in Urinary Glycosaminoglycan (uGAG) Dermatan Sulfate — -50.41; -38.94; -63.49; -51.82 percentage change
Mucopolysaccharidosis VI · Maroteaux-Lamy Syndrome
Primary: Change in Height — 67.3; 80.3; 81.9; 91.3 centimeters
Adrenoleukodystrophy · Metachromatic Leukodystrophy · Globoid Cell Leukodystrophy
Primary: Number of Patients With Donor Cell Engraftment — 26 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
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 · 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
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
Neimann-Pick Disease
Primary: Number of Participants With Tolerabilty of 200 mg Vorinostat in Niemann-Pick Disease, Type C1 — 12 Participants
Sphingomyelin Lipidosis
Primary: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) — 4; 9; 7; 20 Participants
Mucopolysaccharidoses
Primary: Carotid Intima-media Thickness, Measured in Millimeters — 0.44; 0.52; 0.56 mm
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
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)…