30 closest matches · ranked by relevance
Pompe Disease (Late-Onset) · Glycogen Storage Disease Type II (GSD-II) · Glycogenesis Type II
Primary: Summary of Participants Reporting Treatment-Emergent Adverse Events For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600) — 60; 37; 21…
Pompe Disease (Late-onset) · Glycogen Storage Disease Type II (GSD-II) · Acid Maltase Deficiency Disease
Primary: Summary of Patients Reporting Treatment-Emergent Adverse Events — 60; 30; 32; 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
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…
Pompe Disease
Primary: Number of Participants With Adverse Events — 3; 3; 15 Participants
Pompe Disease (Late-Onset) · Glycogen Storage Disease Type II (GSD II) · Glycogenesis 2 Acid Maltase Deficiency
Primary: Change From Baseline in Tissue Glycogen Content in Quadriceps Muscle Biopsy Samples at Week 26 — 5.3; -1.6 percent area occupied by glycogen — p=0.1860
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
Pompe Disease (Late-onset)
Primary: Change From Baseline to Week 52 in 6 Minute Walk Distance (6MWD) — 21.31; 7.10 meter — p=0.048
Glycogen Storage Disease Type II;Pompe's Disease
Primary: PAP: Change From Baseline in Percent Predicted FVC in Upright Position at Week 49 — 2.89; 0.46 percent predicted FVC — p=0.0074
Pompe Disease
Primary: Inspiratory Load Compensation - Inspiratory Volume — 288.41; 554.34; 743.71; 927.57 mL
Pompe Disease
Primary: Number of Participants With Adverse Events. — 5; 5 Participants
Pompe Disease (Late-onset)
Primary: Incidence of Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (TESAEs), and TEAEs Leading to Discontinuation of Study Drug — 80; 37…
Pompe Disease · Glycogen Storage Disease Type II (GSD II)
Primary: Pharmacokinetics (PK): Maximum Observed Plasma Concentration (Cmax) of Alglucosidase Alfa — 204000; 307000 nanograms per milliliter (ng/mL)
Pompe Disease
Primary: Number of Participants With a Change in Creatine Kinase (CK) Reflecting Worsening of Muscle Involvement — 1; 0 participants
Glycogen Storage Disease Type II Pompe Disease
Primary: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (TESAEs), Infusion Associated Reactions (IARs) and…
GNE Myopathy
Primary: Mean Area Under the Curve (AUClast) of Plasma ManNAc (Baseline-adjusted) — 7461; 9432 hr*ng/mL
Duchenne Muscular Dystrophy · Becker Muscular Dystrophy
Primary: Change From Baseline in 6MWD at Week 48 — -41.81; -12.86; -42.56 meters — p=0.4756
Muscular Dystrophy, Duchenne · Muscular Dystrophies · Muscular Disorders, Atrophic
Primary: Change From Baseline in 6MWD at Week 48 — -60.67; -47.69 meters — p=0.213
Duchenne Muscular Dystrophy · Becker Muscular Dystrophy · Dystrophinopathy
Primary: Number of Participants With Treatment Emergent Adverse Events (TEAEs) — 91; 31; 26 Participants
Pompe Disease · Glycogen Storage Disease Type II
Primary: Change From Baseline in Number of Patients With Anti-Recombinant Human Acid Alfa-glucosidase (Anti-rhGAA) Immunoglobulin G (IgG) Antibody at End of Study — 2; 2…
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)…
Sphingomyelin Lipidosis
Primary: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) — 4; 9; 7; 20 Participants
Pompe Disease
Primary: Off-ventilator Spontaneous Breathing (SB) at Baseline and Day 180 — 0.52; 4.72 hours per day
Variant Late-Infantile Neuronal Ceroid Lipofuscinosis
Primary: Incidence And Severity Of Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) — 13; 5; 7; 7 Participants
Jansky-Bielschowsky Disease · Batten Disease · Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2
Primary: Motor-Language (ML) Scale Score During 300 mg Dosing Period — 3.5; 3.1; -0.4 units on a scale — p=0.0002
Lysosomal Acid Lipase Deficiency
Primary: Percentage Of Participants Achieving Alanine Aminotransferase Normalization — 31; 7; 56; 37 percentage of participants — p=0.0271
Gaucher Disease, Type 1
Primary: Change From Baseline to 12 Months in Hemoglobin Concentration for the 60 U/kg Treatment Group. — 2.429 g/dL — p=<0.0001
Pompe Disease
Primary: Incidence of Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (TESAEs), and Adverse Events (AEs) Leading to Discontinuation of Study…
Gaucher Disease, Type 3
Primary: Change From Baseline to 12 Months (Week 53) in Hemoglobin Concentration — 2.15 g/dL
Fabry Disease
Primary: Globotriaosylceramide (GL-3) Clearance in Capillary Endothelium in the Skin — 2; 14; 5; 0 patients