Keratin-associated epidermolysis bullosa simplex: phenotypes and challenges in clinical trials – a narrative review and systematic update

Abstract Introduction Clinical research on innovative therapies for the rare genodermatosis epidermolysis bullosa (EB) faces significant challenges, including small sample sizes, disease heterogeneity with intra- and inter-individual variability, limited understanding of pathogenic mechanisms and na...

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Hlavní autoři:	Verena Wally, Tobias Welponer, Hans-Peter Wiesinger, Anja Diem, Konstantin Thiel, Martin Geroldinger, Georg Zimmermann, Julia I. Hummel, Sonja Dorfer, Josefina Piñón Hofbauer, Johann W. Bauer, Martin Laimer
Médium:	Artigo
Jazyk:	Inglês
Vydáno:	BMC 2025-06-01
Edice:	Orphanet Journal of Rare Diseases
Témata:	Epidermolysis bullosa simplex Blisters Clinical trials Case reports
On-line přístup:	https://doi.org/10.1186/s13023-025-03822-0
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