30 closest matches · ranked by relevance
Barlow's Disease · Prolapse; Mitral
Primary: Cardiac Death — 3.9; 3.8 percentage of participants with event
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
Epidermolysis Bullosa Dystrophica · Epidermolysis Bullosa
Primary: Number of Wounds by Healing Category Per Investigator Visual Assessment — 35; 5; 2; 28 wounds
Papillon-Lefevre Disease
Primary: Serum Concentrations of Interleukin (IL)-1 Beta — 1100.00; 896.21; 65559; 45734 pg/ml
Epidermolysis Bullosa
Primary: Percentage of Participants With Event-free Survival — 67; 80; 50; 29 Percentage of participants
Cerebrotendinous Xanthomatosis (CTX)
Primary: Number of Participants Who Had Positive Genetic Test Results — 4 Participants
Fibrodysplasia Ossificans Progressiva
Primary: Annualized New Heterotopic Ossification (HO) — 9427.1; 23720.2 cubic millimeters (mm^3)
Inflammatory Bowel Disease · Perianal Fistula · Perianal Abscess
Primary: Prevalence of Inflammation as Diagnosed by Capsule Endoscopy — 6 participants
Recessive Dystrophic Epidermolysis Bullosa
Primary: Increased Expression of Full-length Type VII Collagen as Assessed by Immunofluorescence — 31.8; 28.4; 65.6 C7 Expression (percent of normal skin)
Interstitial Lung Disease · Idiopathic Pulmonary Fibrosis
Primary: Number of Participants With ILA (Interstitial Lung Abnormalities) — 85; 13 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
Rheumatologic Disease
Primary: Flow Mediated Dilatation (FMD)-Diameter of Artery — 3.79; 3.78 mm
Barrett's Esophagus
Primary: Change in Ki67 Biomarker Expression — 1539; 1556; 1575; 1864 cells/mm^2
Chronic Obstructive Pulmonary Disease · Emphysema
Primary: Percent Change of Circulating Anti-GRP78 IgG Levels — -17.0; -2.6 % change
Epidermolysis Bullosa
Primary: Event-free Survival — 4; 2; 1 Count of Participants
Systemic Sclerosis
Primary: Change in Modified Rodnan Skin Score (MRSS) — -10; -3.0 units on a scale
Pemphigoid, Bullous
Primary: Number of Participants With Anti-Drug Antibodies — 0 Participants
Adrenoleukodystrophy · Metachromatic Leukodystrophy · Globoid Cell Leukodystrophy
Primary: Number of Patients With Donor Cell Engraftment — 26 Participants
Dupuytren's Disease
Primary: Early Disease: Nodule Hardness at 12 Months — 58.1; 61.2 Durometer Arbitrary Units (AU)
Recessive Dystrophic Epidermolysis Bullosa
Primary: Restoration of Full-length Type VII Collagen as Assessed by Immunofluorescence. — 5; 5; 4; 4 Fluorescence Intensity (MFI) for C7
Relapsing Polychondritis
Primary: Physician Global Assessment of Disease Activity — 21; 14; 12; 43 mm
Ocular Cicatricial Pemphigoid
Primary: Number of Participants With no Evidence of Further Scarring (Fosters Staging) at 16 Weeks — 0 participants
Fibrodysplasia Ossificans Progressiva
Primary: Percentage of Responders at Week 6 — 100; 88.9; 88.9 percentage of subjects — p=0.1664
Barrett Esophagus · Esophageal Carcinoma
Primary: Reversal of Oxidative DNA Damage as Assessed by Changes in 8-hydroxy-2' -Deoxyguanosine (8OHdG) Immunostaining — 1.62 % of strongly/moderately stained nuclei — p=0.54
Barrett's Esophagus · Esophageal Squamous Dysplasia · Esophageal Cancer
Primary: Number of Participants With Complete Eradication of Esophageal Dysplasia — 51 Participants
Behçet's Syndrome
Primary: Area Under the Curve (AUC) for the Number of Oral Ulcers From Baseline Through Week 12 (AUC W0-12) — 222.14; 129.54 Ulcers*days — p=<0.0001
Scleroderma
Primary: Improvement in the Modified Rodnan Skin Score. — 46.75 percent improvement from baseline
Psoriasis
Primary: Change in Histology
Barrett's Esophagus
Primary: Eradication of Barrett's Esophagus — 2 Participants
Epidermolysis Bullosa
Primary: Proportion of Patients With First Complete Closure of the EB Target Wound Within 45 Days of Treatment — 45; 33 Participants — p=0.013