Small molecule drug development for rare genodermatoses - evaluation of the current status in epidermolysis bullosa.
Verena WallyManuela ReisenbergerSophie KitzmüllerMartin LaimerPublished in: Orphanet journal of rare diseases (2020)
We provide a comprehensive summary of the current status of clinical small molecule development for EB and discuss prospects and limitations in orphan drug development for rare conditions like EB.