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Small molecule drug development for rare genodermatoses – evaluation of the current status in epidermolysis bullosa

BACKGROUND: Hereditary epidermolysis bullosa (EB) comprises a heterogeneous group of rare genodermatoses, which are caused by mutations in genes involved in the maintenance of the structural and functional integrity of dermo-epidermal adhesion in various stratified epithelia. In severe variants, gen...

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Detalles Bibliográficos
Publicado en:Orphanet J Rare Dis
Autores principales: Wally, Verena, Reisenberger, Manuela, Kitzmüller, Sophie, Laimer, Martin
Formato: Artigo
Lenguaje:Inglês
Publicado: BioMed Central 2020
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Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC7574495/
https://ncbi.nlm.nih.gov/pubmed/33076941
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-020-01467-9
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