Additional file 2 of Small molecule drug development for rare genodermatoses – evaluation of the current status in epidermolysis bullosa

Additional file 2: Table S1. Clinical trials published for the treatment of EB using small molecules. This table is a summary of published small molecules in the therapy of Epidermolysis bullosa. Publications are ordered by EB subtype and type of trial (RCT, CT, case study), most recent publications on top of each section. The benefit for the patient was clustered into the groups reduction of blister numbers, itch reduction, pain reduction, improvement of wound healing, prevention and treatment of SCCs, and others. Others include QoL (n = 1), relieve anal sphincter spasm and fissuring (n = 1), lower stricture indices in esophageal stenosis (n = 1), plasma phospholipid and fatty acid profiles (n = 1), and reduction of inflammation (n = 1). na: not applicable. * Primary or clinically relevant endpoints are given for RCT trials. # Indicates drugs that are currently under clinical investigation in recruiting or running registered trials (Table S2)