30 closest matches · ranked by relevance
Familial Dysautonomia
Primary: Change From Baseline of Forced Vital Capacity — 5.5; 4.7; 0.9 percentage change from baseline
Menkes Disease · Occipital Horn Syndrome
Primary: Treatment Related Adverse Events as Assessed by CTCAE v4.0 — 10; 6 adverse events
Familial Dysautonomia
Primary: Composite Daily Score — 6.9; 9.7 units on a scale
Dysautonomia, Familial · Baroreflex Failure Syndrome
Primary: Number of Participants Who Reported Adverse Events Related to Study Drug — 0; 0; 0 Participants
Postural Tachycardia Syndrome
Primary: C-peptide Levels After Oral Glucose Tolerance Test — 40.6; 23.2 pg/mL
Familial Amyloid Polyneuropathy · Familial Amyloidosis
Primary: Neurologic Impairment Score + 7 (NIS+7) — 8.2; 26.3; 6.2; 12.5 units on a scale — p=<0.001
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
Heart Failure
Primary: Baseline Heart Rate Recovery — 32.5; 30.4 beats per minute
Familial Hyperlipidemia
Primary: Familial Hypercholesterolemia (FH) Determination for Family Members of School Children With Elevated Cholesterol and Documented FH Variant — 3; 8 participants
Obesity
Primary: Insulin Resistance — 3.8; 3.1 mg/kg/min
Familial Amyloid Polyneuropathy
Primary: Percentage of Participants With Response to Treatment as Measured by Neuropathy Impairment Score - Lower Limb (NIS-LL) at Month 18 — 45.3; 29.5 percentage of participants
Postural Tachycardia Syndrome · Orthostatic Intolerance
Primary: 24 Hour Urinary Sodium Excretion During Treatment Normalized to Creatinine — 140; 132; 137; 154 Milliequivalents per gram (mEq/g)
Symptomatic Neurogenic Orthostatic Hypotension (NOH) · Non-diabetic Neuropathy · Primary Autonomic Failure
Primary: Change in Dizziness/ Lightheadedness/ Feeling Faint/ or Feeling Like You Might Blackout (OHSA Item 1) — 1.3; 1.9 units on a scale — p=0.509
Symptomatic Neurogenic Orthostatic Hypotension (NOH) · Non-diabetic Neuropathy · Primary Autonomic Failure
Primary: Change in Orthostatic Hypotension Questionnaire Score (OHQ) — -1.83; -0.93 units on a scale — p=0.003
Hypertension · Pure Autonomic Failure · Multiple System Atrophy
Primary: Change in Systolic Blood Pressure — 19; 3 mm Hg
Neurogenic Orthostatic Hypotension · Non-Diabetic Autonomic Neuropathy · Multiple System Atrophy
Primary: Change in Orthostatic Hypotension Questionnaire Composite Score (OHQ) — 0.57; 0.90 units on a scale — p=0.438
Postural Orthostatic Tachycardia Syndrome
Primary: 24hr Urinary Sodium — 16.8; 280; 14.7; 245 milliequivalents
Postural Orthostatic Tachycardia Syndrome
Primary: Change in Heart Rate — 17.0; 13.1 beats per minute
Hypoglycemia
Primary: Change in Baroreflex Sensitivity — 2.3; -1.6 ms/mmHg
Transthyretin Familial Amyloidosis Polyneuropathy (TTR-FAP) · Transthyretin Cardiomyopathy (TTR-CM) · Familial Amyloid Cardiomyopathy
Primary: Demographical Characteristics of Participants — 20; 11; 39; 19 participants
Neurofibromatosis 1
Primary: Mitochondrial Respiration Efficiency (as Measured by OCR). — 67.7167; 53.7000; 73.3358; 68.5789 picomole/minute — p=<0.01
Parkinson's Disease
Primary: L-DOPA Insensitivity — 5 Participants — p=0.00468
Postural Tachycardia Syndrome · Autonomic Dysfunction · Postural Orthostatic Tachycardia Syndrome
Primary: The Change in Composite Autonomic Symptom Score (COMPASS-31) — -5.40; -5.94 score on a scale
Healthy Participants
Primary: Plasma Dopa 1 hr After Breakfast — 11670; 1705 pg/mL
Orthostatic Hypertension
Primary: Blood Pressure (Systolic) — 156; 158; 179; 148 mm Hg
Parkinson Disease · Orthostatic Hypotension
Primary: Change in Systolic Blood Pressure From Supine to Tilt at 3 Minutes — -24.22; -20.32 mmHg
Symptomatic Neurogenic Orthostatic Hypotension
Primary: Time To Intervention — 52.00; 42.00; NA; NA Days — p=0.803
Myalgic Encephalomyelitis/Chronic Fatigue Syndrome · Chronic Fatigue Syndrome · Myalgic Encephalomyelitis
Primary: Change in Peak Oxygen Uptake (Peak VO2) Between the First and Second iCPET — 13.3; -40.3; 111.8 mL/min — p=0.043
Familial Amyotrophic Lateral Sclerosis
Primary: Mean Change in SOD1 CSF — 6.8 ng/ml
Hypotension, Orthostatic
Primary: Post-treatment Score For Item 1 of The Orthostatic Hypotension Symptom Assessment (OHSA) Scale — 4.1; 5.2 scores on a scale — p=<0.001