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
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
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 (MPS)
Primary: Number of Treatment Emergent Serious Adverse Events (SAE) — 5; 3; 2 events
Mucopolysaccharidosis IIIB
Primary: Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs) — 1; 0; 1 Participants
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: 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
Adrenoleukodystrophy · Metachromatic Leukodystrophy · Globoid Cell Leukodystrophy
Primary: Number of Patients With Donor Cell Engraftment — 26 Participants
MPS IV A
Primary: Subject Incidence of Treatment Emergent AEs — 18; 18; 17; 17 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
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 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
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
Hurler Syndrome
Primary: Percentage of Adherence to the Scheduled Weekly Infusion by the Participants — 99 percentage
Sphingomyelin Lipidosis
Primary: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) — 4; 9; 7; 20 Participants
Argininosuccinic Aciduria · Amino Acid Metabolism, Inborn Errors · Urea Cycle Disorders
Primary: Measures of Liver Function: AST and ALT — 36.2; 52; 31.7; 57.86 IU/L
Neimann-Pick Disease
Primary: Number of Participants With Tolerabilty of 200 mg Vorinostat in Niemann-Pick Disease, Type C1 — 12 Participants
Hereditary Sensory and Autonomic Neuropathy Type I
Primary: Charcot Marie Tooth Neuropathy Score — 25.67; 20.22 scores on a scale
Mucopolysaccharidosis I
Primary: Number of Participants With Adverse Events — 3; 6; 7; 0 participants
Mucopolysaccharidosis VI
Primary: Change From Baseline in 12-minute Walk Test at 24 Weeks — 109; 26 meters — p=0.025
Mucopolysaccharidosis I · Hurler Syndrome · Hurler-Scheie Syndrome
Primary: Safety Evaluation — 3; 0; 0; 1 participants
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
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
Phelan McDermid Syndrome
Primary: ABC - Social Withdrawal Subscale — 10.31; 4.6; 14.8; 6.2 score on a scale
Spinal Muscular Atrophy
Primary: Safety and Tolerability of Sodium Phenylbutyrate in Neonates and Infants With SMA — 1; 8; 0; 1 Participants
Metachromatic Leukodystrophy (MLD)
Primary: Number of Participants With Treatment-emergent Adverse Events (TEAEs) — 4; 5; 4 participants
Mucopolysaccharidoses
Primary: Carotid Intima-media Thickness, Measured in Millimeters — 0.44; 0.52; 0.56 mm
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…
Phelan-McDermid Syndrome
Primary: Safety and Tolerability — 17; 17; 1; 3 Participants