Items where the Publication is Orphanet journal of rare diseases

Year Authors/Creators Item Type Full Text

Article

Understanding the socioeconomic costs of dystrophic epidermolysis bullosa in Europe: a costing and health-related quality of life study. (2022) Angelis, A; Mellerio, JE; Kanavos, P

Development of the natural history component of an early economic model for primary sclerosing cholangitis. (2025) Bowlus, Christopher; Levy, Cynthia; Kowdley, Kris V; Kachru, Nandita; Jeyakumar, Sushanth; Rodriguez-Guadarrama, Yael; Smith, Nathaniel; Briggs, Andrew; Sculpher, Mark; Ollendorf, Daniel

Quality of life and its contributors among adults with late-onset Pompe disease in China. (2021) Chen, Shanquan; Wang, Jingxuan; Zhu, Jianfeng; Chung, Roger Yat-Nork; Dong, Dong