Increased phototoxic burn tolerance time and quality of life in patients with erythropoietic protoporphyria treated with afamelanotide - a three years observational study.
Jasmin Barman-AksözenMichèle NydeggerXiaoye Schneider-YinAnna-Elisabeth MinderPublished in: Orphanet journal of rare diseases (2020)
Treatment of EPP patients with afamelanotide is highly effective under real-world conditions. We suggest PBTT as a clinical meaningful endpoint in further clinical trials.