30 closest matches · ranked by relevance
Hunter Syndrome
Primary: Infusion-Related Adverse Event (IRAE) Rates Between IgG Anti-idursulfase Antibody Positive (Ab+) and Anti-idursulfase IgG Antibody Negative (Ab-) Patients — 0.0121…
Hunter Syndrome · Mucopolysaccharidosis II · MPS II
Primary: Safety Evaluation — 28; 0; 0; 16 participants
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
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…
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
Hunter Syndrome
Primary: Levels of Total Glycosaminoglycan (GAG) in CSF — 816.750 ng/mL
Mucopolysaccharidosis (MPS)
Primary: Number of Treatment Emergent Serious Adverse Events (SAE) — 5; 3; 2 events
Hunter Syndrome
Primary: Height Overall — 113.759; 108.143 centimeter (cm) — p== 0.011
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
Hurler Syndrome
Primary: Percentage of Adherence to the Scheduled Weekly Infusion by the Participants — 99 percentage
Hunter Syndrome
Primary: Number of Participants Who Were Screened For The Follow-On Study With an Investigational Agent — 17 participants
Hemophagocytic Syndrome (HPS)
Primary: Overall Survival at 2 Months — 6 Participants
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
Primary: Number of Participants With Treatment-emergent Adverse Events (TEAEs) — 4; 10; 5 Participants
X-Linked Retinitis Pigmentosa
Primary: Number of Participants Meeting the Primary Outcome Defined as Any of the Below Events Occurring During the 9 Weeks Following Administration, at Least Possibly Related to…
Urea Cycle Disorders
Primary: Rate of Adverse Events (Number of Participants Who Experienced Any AE Considered Related to Study Drug) — 33 participants
Phelan McDermid Syndrome
Primary: ABC - Social Withdrawal Subscale — 10.31; 4.6; 14.8; 6.2 score on a scale
Primary Immunodeficiency Diseases (PID)
Primary: Safety: Number of Participants With Any Severe Related Treatment-emergent Adverse Events (TEAEs) Per Infusion (Excluding Infections) — 1; 0 Participants
Hunter Syndrome
Primary: Number of Serious Adverse Event (SAE) — 0; 8; 3; 3 events
Hemophagocytic Lymphohistiocytosis · Chronic Active Epstein-Barr Virus Infection · Chronic Granulomatous Disease
Primary: Percentage of Participants With Overall Survival (OS) — 80.4; 66.7 percentage of participants
Short Stature
Primary: Height Standard Deviation Score — -2.52; -1.57; -1.19; -1.09 standard deviation score
Hereditary Angioedema · Angioneurotic Edema · Genetic Disorders
Primary: Time to Beginning of Relief of Symptoms — 62; 508; 61 minutes — p=0.003
Treatment Resistant Nephrotic Syndrome
Primary: Significant Reduction in Proteinuria to Remission Levels During the Treatment Period (Includes Complete and Partial Remission, as Defined in the Outcome Measure…
Urea Cycle Disorders
Primary: Venous Ammonia Levels at the Peak and Mean TNUAC Time-normalized Area Under the Curve) — 79.1; 56.3; 38.4; 26.5 μmol/L
Eosinophilia · Hypereosinophilic Syndrome · Leukocyte Disorder
Primary: Mean Percent Change in Eosinophil Count After Glucocorticoid Challenge — -69.6; -13.6 percentage of eosinophil count
Wiskott-Aldrich Syndrome (WAS)
Primary: Safety of Reduced Conditioning Regimen — 8 Participants
Hypoparathyroidism
Primary: Serum Calcium Level — 2; 1.92 mmol/liter
Severe Hemophilia A
Primary: Number of Participants Who Developed Target Joint Bleeding — 17 Participants
Hypoparathyroidism
Primary: Number of Participants With Treatment Emergent Adverse Event (TEAE) and Treatment Emergent Serious Adverse Event (TESAE) — 13; 48 Participants
Prader-Willi Syndrome
Primary: Body Composition — 51.2; 48.5; 52.1 percentage of body fat — p=<0.05