Affiliation: | 1. Actelion Pharmaceuticals Ltd, Allschwil, Switzerland;2. Numerus Ltd, Wokingham, UK;3. Academic Medical Center, Amsterdam, the Netherlands;4. Regional Coordinator Centre for Rare Diseases, Azienda Ospedaliera Santa Maria della Misericordia di Udine, Udine, Italy;5. Royal Free and University College Medical School, London, UK |