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Characteristics of Patients With Recessive Dystrophic Epidermolysis Bullosa
Recessive dystrophic epidermolysis bullosa (RDEB) is a disease caused by genetic mutations in the gene for type VII collagen. Patients with RDEB develop large,…
A Phase II, Closed Label, Placebo Controlled, Randomized, Double-Blinded Clinical Trial to Evaluate the Efficacy and Safety of TolaSure Gel, 5% w/w Targeting Aggregated Mutant Keratin in Epidermolysis Bullosa Simplex (TAMES)
This Phase II clinical study will assess the efficacy, safety and tolerability of topical TolaSure Gel in adults and pediatric patients (4 years of age and old…
Mesenchymal Stem Cell Extracellular Vesicles for the Treatment of Recessive Dystrophic Epidermolysis Bullosa Wounds
INVESTIGATIONAL PRODUCT: AGLE-102 is an allogeneic extracellular vesicle (EV) product derived from normal donor mesenchymal stem cells (MSCs). INDICATION AND …
A Pilot Study of the Restoration of Functional Laminin 332 in JEB Patients With Nonsense Mutations After Topical and Intravenous Gentamicin Treatment
Herlitz junctional epidermolysis bullosa (H-JEB), an incurable, fatal, inherited skin disease, is caused by loss-of-function mutations in the LAMA3, LAMB3 or L…
Targeting Collagen VII Antibodies With IV IgG in Dystrophic Epidermolysis Bullosa
The study objective is to see if IV IgG treatment in Recessive Dystrophic Epidermolysis Bullosa (RDEB) skin in conjunction with VYJUVEK treatment improves woun…
A Prospective, Observational, Long-Term Follow-Up (LTFU) Study to Evaluate the Long-Term Safety of the Krystal Biotech, Inc. Gene Therapy Backbone Products Using HSV-1 Backbone
The main objective of this prospective, observational, long-term follow-up (LTFU) study is to evaluate the long-term safety profile of the gene therapy product…
A Natural History Study of Corneal Abrasions in Patients With Dystrophic Epidermolysis Bullosa (DEB)
This study is a non-interventional, observational study that will evaluate the natural history of corneal abrasions in patients with Dystrophic Epidermolysis B…
Targeting Collagen VII Antibodies in Bullous Diseases Using Efgartigimod IV (VYVGART)
The study objective is to see if IV Efgartigimod and Vyjuvek treatment in Recessive Dystrophic Epidermolysis Bullosa (RDEB) and IV Efgartigimod treatment in Ep…
A Double-Blind Crossover of KB803 and Matched Placebo for the Treatment and Prevention of Corneal Abrasions in Dystrophic Epidermolysis Bullosa
KB803-EYE-01 is a Phase 3 double-blind, randomized, placebo-controlled, crossover study to evaluate the safety and efficacy of KB803 versus matched placebo in …