30 closest matches · ranked by relevance
Alzheimers Disease · Dementia · Alzheimers Disease, Familial
Primary: Assess Cognitive Efficacy in Individuals With Mutations Causing Dominantly Inherited AD as Measured by the DIAN-Multivariate Cognitive Endpoint (DIAN-MCE); — 1.063…
ADNP Syndrome
Primary: Number of Participants With an Adverse Event — 9 Participants
Pantothenate Kinase-Associated Neurodegeneration
Primary: Number of Treatment-emergent Adverse Events Assessed Using CTCAE v4.0 — 1.15; 1.38; 1.14; 0.86 Events per person — p=0.50
Obesity
Primary: Adipose Tissue Gene Expression of UCP1 (Uncoupling Protein 1)- Differences by Obesity Status — 7.79; 24.33 fold change — p=<0.01
Autosomal Dominant Hypocalcemia (ADH)
Primary: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs) — 3; 1 participants
Pseudohypoparathyroidism · Albright Hereditary Osteodystrophy
Primary: Intelligence Quotient — 85.9; 104.3; 103.2 units on a scale
Prader-Willi Syndrome
Primary: Change in Weight — -.5 kg — p=.07
Mild Cognitive Impairment · Dementia; Alzheimer's Type (Etiology)
Primary: Participant Interest in PET Biomarker Disclosure — 73 Participants
Healthy · Lean
Primary: Changes in NT-proANP From Baseline to 8 Hours — -0.33 nmol/l
Down Syndrome · Amyloid Beta-protein
Primary: Sensitivity of the Independent Visual Assessment of Detecting Cerebral Amyloid-beta in Individuals With Down Syndrome and Specificity in Subjects Without Down Syndrome…
22q11 Deletion Syndrome
Primary: Safety and Tolerability of NB-001 — 18; 24; 14; 20 Participants
Obesity · Insulin Resistance · Diabetes Mellitus Type 2
Primary: Serum Apelin Levels — 1.45; 1.91 ng/ml — p=0.001
Down Syndrome · Obstructive Sleep Apnea Syndrome
Primary: Change in Epworth Sleepiness Scale From Baseline to End of Study — -1; -0.5; 1 units on a scale
Alzheimer Disease
Primary: ROC AUCs (MCI/AD vs Cognitively Unimpaired (CU)) for Medial Temporal Lobe (MTL) UCB-J Distribution Volume Ratio (DVR) and Hippocampal Volume — 0.79; 0.81 probability…
Neurogenic Orthostatic Hypotension
Primary: Change in the OHQ (Orthostatic Hypotension Questionnaire) Composite Score — 4.61; 4.17; 3.35; 3.5 Score on a scale
Short Stature
Primary: Height Standard Deviation Score — -2.52; -1.57; -1.19; -1.09 standard deviation score
Prader-Willi Syndrome
Primary: Hyperphagia Questionnaire (HQ-CT) Change From Baseline at Visit 7 (Week 13) — -5.94; -4.27 score on a scale — p=0.1983
Growth Hormone Deficiency · Septo-Optic Dysplasia · Hypopituitarism
Primary: Change in Anthropometric Measures Over Time — 1.97; -0.61; 1.39; 1.22 Z-score
Attention Deficit Hyperactivity Disorder
Primary: Change From Baseline in Total AISRS Score at the End of Double-blind Treatment (Week 8) — -17; -17 units on a scale
Down Syndrome
Primary: Incidence of Adverse Events (TEAEs) — 5; 2; 0 participants
Type1 Diabetes
Primary: Percent of Time-in-Range (TIR) on NAP Versus UMPC. — 86.08; 87.25 Percent of time between 70-180 mg/dl — p=0.2
Phelan-McDermid Syndrome
Primary: Safety and Tolerability — 17; 17; 1; 3 Participants
Angelman Syndrome · Nervous System Diseases
Primary: Average Change in Functioning in Specific Areas of Development, Including Speech and Communications Skills, Cognitive Abilities and Daily Living Skills — 5.7; 4.7; 5.8…
Down Syndrome · Intellectual Disability
Primary: Efficacy of the Drug Memantine as Assessed by Change in Score on the California Verbal Learning Test-II (CVLT-II) Short Form Total Free Recall — 3.3; 3.49 score on a…
Down Syndrome
Primary: Changes in Neuropsychological Measures From Baseline to End of Study — -0.22; -0.84; 0.56; 0 units on a scale — p=0.403
Peroxisome Biogenesis Disorders
Primary: Peroxisome Biochemical Functions as Measured by Plasma Very Long Chain Fatty Acid — 0.180; 0.188 ratio
Pitt Hopkins Syndrome
Primary: Safety and Tolerability — 15; 0; 4; 12 Participants
Alzheimer's Disease
Primary: Symptoms of Agitation as Measured by the Pittsburgh Agitation Scale — 7.14; 5.55; 5.69; 4.97 score on a scale — p=.015
Alzheimer's Disease
Primary: Amyloid Deposition Obtained on a 18F-flutemetamol Brain Scan. — 1.65 ratio of flutemetamol absorbed — p=<0.05
Genetic Disorder · Noonan Syndrome
Primary: Change in Height SDS (Japanese National Reference Data) — 0.84; 1.47 Standard deviation score — p=< 0.0001