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
Congenital Sucrase-isomaltase Deficiency (CSID)
Primary: Prevalence of CSID Genetic Variants — 27; 0 Participants
Congenital Ichthyosis
Primary: Number of Participants With Adverse Events (AEs) in Part 1 of Trial (Weeks 0-8) — 7; 7; 5; 5 participants
Thalassemia · Sickle Cell Disease · Diamond Blackfan Anemia
Primary: Safety Profile of Deferasirox Based Upon Drug Administration and Reporting of Serious Adverse Events — 0; 0; 1; 4 Participants
Urea Cycle Disorders
Primary: Mortality — 18.4; 20.7 participant per 1000 person-years
Congenital Hearing Loss
Primary: Number of Participants Who do Not Receive Diagnostic Audiological Testing (Aim 1) — 15; 8 Participants
Molybdenum Cofactor Deficiency, Type A
Primary: Overall Survival — 3 Participants
Cerebrotendinous Xanthomatosis (CTX)
Primary: Number of Participants Who Had Positive Genetic Test Results — 4 Participants
Adrenoleukodystrophy · Metachromatic Leukodystrophy · Globoid Cell Leukodystrophy
Primary: Number of Patients With Donor Cell Engraftment — 26 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
Pseudohypoparathyroidism · Albright Hereditary Osteodystrophy
Primary: Intelligence Quotient — 85.9; 104.3; 103.2 units on a scale
Respiratory Distress Syndrome, Newborn
Primary: Mortality — 5; 3; 5 Participants
Congenital Myotonic Dystrophy
Primary: Change in Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS) — -1.65; -3.40 score on a scale — p=0.0514
Congenital Diaphragmatic Hernia
Primary: Proportion of Infants Who Are Intubated Prior to Umbilical Cord Clamping — 17 Participants
Plagiocephaly
Primary: Safety of Use of the Premie Pouch in VLBW Infants — 0 participants
Dyskeratosis Congenita · Aplastic Anemia
Primary: Incidence of Neutrophil Engraftment — 100; 100; 96; 100 Percentage of participants
Congenital Heart Disease · Cardiovascular Disease
Primary: BNP Levels — 16.3; 42.4; 384.3 pg/ml
Factor XIII Deficiency
Primary: Adverse Events — 42; 2; 2 participants
Congenital Disorder
Primary: Feasibility of Pacemaker Implant With Total Thoracoscopic Approach to Epicardial Pacing Lead — 100 percentage of particiapants
Urea Cycle Disorder
Primary: Liver Stiffness as Measured by Shear Wave Elastography — 13; 14 Participants
Sickle Cell Trait
Primary: Acceptability — 11; 8; 11; 8 Participants
Factor XIII Deficiency
Primary: The Incidence of Spontaneous Bleeding Events Requiring Treatment (Treatment is Defined as Administration of a FXIII-Containing Product to Treat the Bleeding Event) — 0…
Mucopolysaccharidosis · Hurler Syndrome · Hunter Syndrome
Primary: Number of Patients With Donor Derived Engraftment — 42 Participants
Down Syndrome
Primary: Step Width — 10.4 cm
Congenital Heart Disease
Primary: Acetaminophen Serum Concentrations — 19; 17; 13; 13 mcg/mL
Non-Neoplastic Hematologic and Lymphocytic Disorder
Primary: Preliminary Efficacy — 83; 10 Participants
Cytomegalovirus Infection
Primary: Number of Ears That Had (1) Improved Hearing or no Change in Hearing (2) Worsened Hearing. — 27; 20; 1; 6 Ears — p=0.0859