30 closest matches · ranked by relevance
MELAS · LHON · Leigh Syndrome
Primary: SAD: Change From Baseline in ECG Results by Timepoint: Corrected QT Interval According to Fridericia's Formula (QTcF) — 386.2; 395.0; 385.8; 399.3 msec
Inherited Mitochondrial Disease, Including Leigh Syndrome
Primary: Change From Baseline in Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Sections I-IV — -0.3; 0.1; -0.6; 0.0 units on a scale — p=0.1875
Inherited Mitochondrial Disease
Primary: Number of Participants With Treatment-emergent Adverse Events (TEAEs) — 91 Participants
Mitochondrial Diseases
Primary: McMaster Gross Motor Function (GMFM 88) — -0.002; -0.12 units on a scale — p=0.95
MItochondrial Myopathies
Primary: Change of Peak Workload (in Watts/kg) During Exercise Testing — 0.0090; 0.020; -0.0300; 0.1050 Watts/kg — p=0.7321
Neurofibromatosis 1
Primary: Mitochondrial Respiration Efficiency (as Measured by OCR). — 67.7167; 53.7000; 73.3358; 68.5789 picomole/minute — p=<0.01
Mitochondrial Myopathy
Primary: Change in Distance Walked (Meters) on the 6-minute Walk Test (6MWT) — 13.5; 36.5; 64.5; 20.4 meters
Mitochondrial Disease · Neurology · Myopathy
Primary: Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Part I-III, Baseline — 23.9; 20.4 Units on a scale
Primary Mitochondrial Disease
Primary: Distance Walked on the 6-minute Walk Test (6MWT) by Visit — 394.1; 378.2 meters
MELAS Syndrome
Primary: Clinical Safety of TIV Vaccine — 4; 0; 0; 0 Participants
Adrenoleukodystrophy · Metachromatic Leukodystrophy · Globoid Cell Leukodystrophy
Primary: Number of Patients With Donor Cell Engraftment — 26 Participants
Hemodialysis-Induced Symptom · Mitochondrial Diseases
Primary: Phosphocreatine (PCR) Recovery Time After Knee Extension Assessed by 31 Phosphorus Magnetic Resonance Spectroscopy (31P-MRS) — 60.59; 62.66 seconds
Primary Mitochondrial Myopathy
Primary: Change in Distance Walked During a 12 Minute Walk Test — 26.75; 30.89 meters
Mitochondrial Diseases
Primary: Average TSST-induced Elevation in Cortisol — 37.44; 33.86; 42.83; 45.42 ng*min/mL
Mucopolysaccharidosis · Hurler Syndrome · Hunter Syndrome
Primary: Number of Patients With Donor Derived Engraftment — 42 Participants
Peripheral Arterial Disease · Peripheral Artery Disease
Primary: Endothelial Function — 6.13; 3.78 % dilation
Fibromyalgia · Mitochondrial Diseases · Movement Disorders
Primary: Heart Rate Variability (Time Domain) — 0.92; 0.83 msec/msec — p=0.77
Healthy · Obesity · Insulin Sensitivity
Primary: Mitochondrial Function in Circulating Cells and Muscle Tissue in Young Women — 26.48; 30.46; 120.04; 140.43 pmol O2/min per 400K cells
Cancer · Skin Fibroblasts · Muscle Weakness
Primary: Change in PCr Recovery Tc Measurement From Baseline to 12 Week NR Supplementation Using the 31P-MRS Skeletal Muscle Submaximal Exercise. — 60; 73 Seconds
MELAS Syndrome
Primary: Mean Change in Cerebral Lactate Concentration (as Measured by Magnetic Resonance Spectroscopy) — -0.09; 0.16; -0.49 IU
Autism Spectrum Disorder
Primary: Change in Mitochondrial Activity in Study Patients: Citrate Synthase — 40.56; 29.09; 27.41; 37.78 nmoles/min/mg protein
Leber's Hereditary Optic Neuropathy
Primary: Number of Treatment Related Adverse Events — 1; 1; 1; 5 Participants
Kinky Hair Syndrome
Primary: Gross Motor Development at 36 Mos of Age or at Death (Mos) — 13.743; 2.455; 15.667 Other - months
Acute Myeloid Leukemia · Myelodysplastic Syndrome
Primary: Number of Subjects With Unacceptable Toxicity — 1 Participants
Lysosomal Storage Disease · Peroxisomal Disorder
Primary: Donor (Allogeneic) Hematopoietic Engraftment — 1 participants
Metachromatic Leukodystrophy (MLD)
Primary: Number of Participants With Treatment Emergent Adverse Events (TEAEs) by Type and Severity — 6; 6; 6; 6 Participants
Neuromuscular Disease
Primary: Urine 15-F2t Isoprostane Concentration — 2.7; 2.6 ng/mg Cr — p=0.88
Severe Combined Immunodeficiency
Primary: Number of Participants With Adverse Events — 10; 9; 0; 10 participants
Bipolar Depression
Primary: The 25-Item Hamilton Depression Rating Scale. — 23.3; 22.1; 17.5; 18.5 units on a scale
Abdominal Obesity · Insulin Resistance · Hypertriglyceridemia
Primary: Change From Baseline in Phosphocreatine Recovery (ViPCr) at 6-months — 1.7; 1.6 mM/s — p=0.97