30 closest matches · ranked by relevance
Pseudoxanthoma Elasticum
Primary: Number of Participants With Treatment-emergent Adverse Events (TEAEs) in Participants Receiving DS-1211b — 8; 7; 6; 6 Participants
Pseudoxanthoma Elasticum
Primary: Von Kossa Staining Per Unit Area of Dermis — -0.0033; 0.0010; 0.0087; 0.0123 microns
Erythropoietic Protoporphyria · EPP · X-Linked Protoporphyria
Primary: The Hospital Anxiety and Depression Scale (HADS) — 4.6; 1.9 score 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
Peroxisome Biogenesis Disorders
Primary: Peroxisome Biochemical Functions as Measured by Plasma Very Long Chain Fatty Acid — 0.180; 0.188 ratio
Cerebrotendinous Xanthomatosis (CTX)
Primary: Number of Participants Who Had Positive Genetic Test Results — 4 Participants
Pseudohypoparathyroidism · Albright Hereditary Osteodystrophy
Primary: Intelligence Quotient — 85.9; 104.3; 103.2 units on a scale
Erythema Multiforme
Primary: Erythema Multiforme Flares on Medication — 1.7 Number of flares
Idiopathic Pulmonary Fibrosis
Primary: EGCG PK Level in Healthy Volunteers — NA; NA; NA; 861 nM
Epidermolysis Bullosa Dystrophica · Epidermolysis Bullosa
Primary: Number of Wounds by Healing Category Per Investigator Visual Assessment — 35; 5; 2; 28 wounds
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
Primary Biliary Cholangitis · Cardiovascular Abnormalities
Primary: The Incidence of Cardiac Events — 0; 0; 0; 0 Participants — p=0.001
Erythropoietic Protoporphyria
Primary: Percent Change From Baseline in Whole Blood Metal-free PPIX Levels — 8.07; -21.48; -41.74 Percent Change
Hyperlipidemia
Primary: Myocardial Perfusion at Rest — 55; 135 IU/s
Psoriasis · Cardiovascular Diseases
Primary: Change in Total Vascular Inflammation of the Aorta as Measured by FDG-PET/CT Between Baseline and Week 16. — -0.017 ratio — p=0.612
Peyronie's Disease
Primary: Average Percent Change of Penile Curvature in Degrees — -21.73; 2.429 percent change — p=0.5154
Palmoplantaris Pustulosis
Primary: Percentage Change in Palmoplantar Pustulosis Severity Index (PPPASI) Before Crossover — 30.1; 7.1 Percentage change
Pyoderma Gangrenosum
Primary: The Proportion of Subjects Achieving 2-point Reduction in the 5-point Investigator Global Assessment (IGA) for the Target Ulcer at Week 12 — 0 Participants — p=1
Pompe Disease (Late-Onset) · Glycogen Storage Disease Type II (GSD-II) · Glycogenesis Type II
Primary: Summary of Participants Reporting Treatment-Emergent Adverse Events For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600) — 60; 37; 21…
Erythropoietic Protoporphyria
Primary: Photosensitivity, Assessed by Measuring the Number of Minutes of Sun Tolerance — 65.8 minutes
Rosacea · Papulopustular Rosacea
Primary: Improvement of the Inflammatory Lesions (Papules, Pustules, Nodules), Erythema, and Telangiectasia of Rosacea as Assessed by the Investigator's Global Assessment (IGA)…
Erythropoietic Protoporphyria · EPP · X-linked Protoporphyria
Primary: Change in Erythrocyte Protoporphyrin Levels — 2314.6; 2240.4 µg/dL
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…
Surgical Adhesions
Primary: TAM2 — 108; 121 Degrees — p=0.8861
Advanced Malignant Mesothelioma · Epithelial Mesothelioma · Recurrent Malignant Mesothelioma
Primary: Objective Tumor Response Rate According to the Response Evaluation Criteria in Solid Tumors (RECIST) Committee — 0 percentage of participants
Epidermolysis Bullosa
Primary: Blister/Erosion Reduction Based on Change in Body Surface Area (BSA) Coverage — 57.25; 39.40; 27.25; 25.45 percentage of BSA involvement
Cystic Fibrosis · Exocrine Pancreatic Insufficiency
Primary: Percentage of Participants Who Were Responders After 1 Week of Treatment With Study Medication — 68.4 percentage of participants
Epidermolysis Bullosa Simplex · Epidermolysis Bullosa Simplex Kobner · Weber-Cockayne Syndrome
Primary: Foot Health Status Questionnaire, Foot Function Domain Score — 61.7; 49.3; 60.2; 56.3 score on a scale
Pseudohypoparathyroidism Type 1a · Albright Hereditary Osteodystrophy
Primary: Change in Urine cAMP — 3048 fm/uL
Macular Degeneration · Wet Macular Degeneration
Primary: Anatomic Response