4 Department of Electrical, Computer, and Systems Engineering (ECSE), Case Western Reserve University, Cleveland, OH, USA.
5 National Center for Advancing Translational Sciences, National Institutes of Health, Bethesda, MD, USA.
6 Orphan Medicines Office, European Medicines Agency, Amsterdam, Netherlands.
7 Office of Orphan Products Development, Food and Drug Administration, Silver Spring, MD, USA.
8 Hyman, Phelps & McNamara, Washington, DC, USA.
9 MDUK Oxford Neuromuscular Centre, Department of Physiology, Anatomy and Genetics, University of Oxford, Oxford, UK.
10 Hitachi Chemical Regenerative Medicine Business Sector, Allendale, NJ, USA.
11 Translational Informatics Division, Department of Internal Medicine, University of New Mexico Albuquerque, Albuquerque, NM, USA.
12 UNM Comprehensive Cancer Center, University of New Mexico Health Science Center, Albuquerque, NM, USA.
13 School of Pharmaceutical Sciences, Institute of Pharmaceutical Sciences of Western Switzerland, University of Geneva, Geneva, Switzerland. marco.prunotto@unige.ch.