First topical gene therapy treatment for dystrophic epidermolysis bullosa

EMA has recommended granting a marketing authorisation in the European Union (EU) for Vyjuvek (beremagene geperpavec) to treat wounds in patients of all ages with dystrophic epidermolysis bullosa (DEB). Dystrophic epidermolysis bullosa is a serious, ultra-rare genetic blistering disease caused by mutations in the collagen type VII alpha 1 chain (COL7A1) gene. This gene is involved in the production of type VII collagen (COL7), a protein crucial in supporting and structuring the layers of the...

Publication Details

Journal
Industrias Culturais (Universidade de Coimbra)
Published
2025-05-09
DOI
https://doi.org/10.58079/13wg2
Primary Topic
Skin and Cellular Biology Research
Type
article
Field-Weighted Citation Impact
0.00
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article

First topical gene therapy treatment for dystrophic epidermolysis bullosa

Industrias Culturais (Universidade de Coimbra)
Skin and Cellular Biology Research
article

First topical gene therapy treatment for dystrophic epidermolysis bullosa

article en

Abstract

EMA has recommended granting a marketing authorisation in the European Union (EU) for Vyjuvek (beremagene geperpavec) to treat wounds in patients of all ages with dystrophic epidermolysis bullosa (DEB). Dystrophic epidermolysis bullosa is a serious, ultra-rare genetic blistering disease caused by mutations in the collagen type VII alpha 1 chain (COL7A1) gene. This gene is involved in the production of type VII collagen (COL7), a protein crucial in supporting and structuring the layers of the...

Industrias Culturais (Universidade de Coimbra)
Good health and well-being
Openalex Percentile: Top 55%
Skin and Cellular Biology Research
DOIOpen Access PDFOpenAlex
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First topical gene therapy treatment for dystrophic epidermolysis bullosa · Industrias Culturais (Universidade de Coimbra) (2025) | TGRS Research Map | TGRS