30 closest matches · ranked by relevance
Congenital Pediatric Disorders
Primary: Overall Survival at 100 Days After Umbilical Cord Blood Transplant in Pediatric Patients. — 0.947 probability of overall survival
Peroxisome Biogenesis Disorders
Primary: Peroxisome Biochemical Functions as Measured by Plasma Very Long Chain Fatty Acid — 0.180; 0.188 ratio
Brain Diseases, Metabolic, Inborn · Urea Cycle Disorder · Ornithine Transcarbamylase Deficiency
Primary: Concentration of Glutamine and Myoinositol by MRS — 4.97; 3.66; 3.78; 4.50 mM — p=0.003
Congenital Heart Disease
Primary: To Evaluate the Incidence of the Pre-proendothelin SNP at Nucleotide 5665 — 10 participants
Respiratory Distress Syndrome, Newborn
Primary: Mortality — 5; 3; 5 Participants
Down Syndrome
Primary: Step Width — 10.4 cm
Congenital Diaphragmatic Hernia
Primary: Proportion of Infants Who Are Intubated Prior to Umbilical Cord Clamping — 17 Participants
Congenital Ichthyosis
Primary: Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) — 7; 7; 5; 5 participants
Congenital Hearing Loss
Primary: Number of Participants Who do Not Receive Diagnostic Audiological Testing (Aim 1) — 15; 8 Participants
Adrenoleukodystrophy · Metachromatic Leukodystrophy · Globoid Cell Leukodystrophy
Primary: Number of Patients With Donor Cell Engraftment — 26 Participants
Congenital Disorder
Primary: Feasibility of Pacemaker Implant With Total Thoracoscopic Approach to Epicardial Pacing Lead — 100 percentage of particiapants
Respiratory Distress Syndrome
Primary: Respiratory Distress Syndrome — 52; 3 participants
Urea Cycle Disorders
Primary: Mortality — 18.4; 20.7 participant per 1000 person-years
Cerebrotendinous Xanthomatosis (CTX)
Primary: Number of Participants Who Had Positive Genetic Test Results — 4 Participants
Sickle Cell Trait
Primary: Acceptability — 11; 8; 11; 8 Participants
Sepsis · Congenital Diaphragmatic Hernia · Neonatal Cardiopulmonary Failure
Primary: TNF-alpha, Baseline — 8.88 pg/mL
Lupus Erythematosus, Systemic · Atrioventricular Block · Sudden Death
Primary: Presence of Conduction Disorders in Patients With SLE Regarding to Its Serologic Profile — 89.22; 91.41; 145.68; 146.72 msec
Plagiocephaly
Primary: Safety of Use of the Premie Pouch in VLBW Infants — 0 participants
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
Mucopolysaccharidosis · Hurler Syndrome · Hunter Syndrome
Primary: Number of Patients With Donor Derived Engraftment — 42 Participants
Urea Cycle Disorder
Primary: Liver Stiffness as Measured by Shear Wave Elastography — 13; 14 Participants
Non-Neoplastic Hematologic and Lymphocytic Disorder
Primary: Preliminary Efficacy — 83; 10 Participants
Pseudohypoparathyroidism · Albright Hereditary Osteodystrophy
Primary: Intelligence Quotient — 85.9; 104.3; 103.2 units on a scale
Non Malignant Disorders · Immunodeficiencies · Congenital Marrow Failures
Primary: Determine the Feasibility of Attaining Acceptable Rates of Donor Cell Engraftment (>25% Donor Cells at 180 Days) Following RIC Regimens in Children < 21 Years Receiving…
Congenital Heart Disease · Cardiovascular Disease
Primary: BNP Levels — 16.3; 42.4; 384.3 pg/ml
Hemangioma
Primary: MRI/MRA of Head/Neck/Chest. — 357 participants
Congenital Heart Block · Neonatal Lupus · Autoantibody-Associated Heart Block
Primary: Recurrence of Advanced Heart Block — 5 Participants
Congenital Heart Defects
Primary: Transition Readiness Assessment Questionnaire (TRAQ) Score — 2.77; 2.95; 3.80; 3.92 units on a scale
Congenital Heart Disease · Periventricular Leucomalacia · Brain Development
Primary: Motor Scale of the Bayley Scales of Infant and Toddler Development-III — 90.0; 87.5; 88.7 score on a scale
Congenital Heart Disease
Primary: Acetaminophen Serum Concentrations — 19; 17; 13; 13 mcg/mL