25 closest matches · ranked by relevance
Inherited Erythromelalgia
Primary: Average Pain Intensity Numerical Rating Scale (PI-NRS) Score - From 0 to 4 Hours Post-dose — 2.08; 2.73; 1.95; 2.04 Units on a scale
Primary Erythromelalgia · Inherited Erythromelalgia
Primary: Average Daily Use of Cooling for Erythromelalgia-Related Pain in Treatment Period 2 — 0.24; 2.4 cooling uses/day
Erythromelalgia
Primary: Carbamazepine Affects Pain in Patients With S241T NaV1.7 IEM Mutation — 3015; 136; 6360; 915 minutes
Erythropoietic Protoporphyria · EPP · X-Linked Protoporphyria
Primary: The Hospital Anxiety and Depression Scale (HADS) — 4.6; 1.9 score on a scale
Erythema Multiforme
Primary: Erythema Multiforme Flares on Medication — 1.7 Number of flares
EPP · XLP
Primary: Change From Baseline in Average Daily Sunlight Exposure Time (Minutes) to First Prodromal Symptom (Burning, Tingling, Itching, or Stinging) Associated With Sunlight…
Blood Platelet Disorders
Primary: Response to Drug Based on Platelet Count at the End of Therapy — 91.6 percentage of participants
Charcot Marie Tooth Disease
Primary: Number of Participants With a Decrease in Cramp Duration — 2; 0 participants
BRAF V600E Mutation
Primary: Number of Participants With Partial Response to Dabrafenib and Trametinib — 4 Participants
Erythropoietic Protoporphyria (EPP)
Primary: Change From Baseline in Average Daily Time (Minutes) to First Prodromal Symptom Associated With Sunlight Exposure Between Hour Post Sunrise and 1 Hour Pre-Sunset at Week…
Erythropoietic Protoporphyria · EPP · X-linked Protoporphyria
Primary: Change in Erythrocyte Protoporphyrin Levels — 2314.6; 2240.4 µg/dL
Atherosclerosis
Primary: Carotid Artery Stiffness (Expressed by Beta-stiffness Index ) in JAK2 V617F Positive ET Patients in Comparison to Healthy Control Subjects in a 4-year Period (From…
Mitochondrial Disease · Neurology · Myopathy
Primary: Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Part I-III, Baseline — 23.9; 20.4 Units on a scale
Parkinson's Disease · Levodopa
Primary: Gastric Emptying Time — 105; 180 minutes — p=0.036
Erythropoietic Protoporphyria
Primary: Time in Direct Sunlight Between 10:00-15:00 on Pain-free Days — 8.25; 0.75 Hours
Erythropoietic Protoporphyria
Primary: Duration of Direct Sunlight Exposure Between 10:00 and 18:00 Hours on Days When no Pain Was Experienced (Pain Score of 0). — 69.4; 40.8 hours
Erythropoietic Protoporphyria
Primary: Cumulative Number of Days of Phototoxic Reactions (Study Efficacy Population) — 39; 45; 129; 179 Days
Myotonia · Non-Dystrophic Myotonia
Primary: Patient-reported Stiffness on the IVR — 2.53; 4.21; 1.60; 5.27 units on a scale — p=<0.001
Fragile X Syndrome
Primary: Safety and Tolerability - Number of Treatment-Emergent Adverse Events — 2; 1; 1; 3 Number of Adverse Events
Epidermolysis Bullosa Dystrophica · Epidermolysis Bullosa
Primary: Number of Wounds by Healing Category Per Investigator Visual Assessment — 35; 5; 2; 28 wounds
Myoclonus Epilepsies, Progressive
Primary: Number of Participants With Treatment-Related Adverse Events After Single Dose Treatment of AUT00201 Compared to Placebo — 5; 5 Participants
Atopic Dermatitis · Ichthyosis Vulgaris
Primary: Heterozygous for Filaggrin (FLG) Null Mutations — 1; 4 participants
Telangiectasia, Hereditary Hemorrhagic
Primary: Change From Baseline Epistaxis Severity Score — -1; -1.1; -1.3; -1.2 units on a scale
Polymorphic Light Eruption
Primary: Severity of PLE Related Pruritus Recorded Using an 11-point Likert Scale — 0.25; 0.47 score on a scale
RYR-1 Myopathy
Primary: Number of Participants Experiencing Adverse Events When Treated With S48168 (ARM210) — 0; 0; 0; 0 Participants