Editor in Chief:
M. Ian Phillips, Director of the Center for Rare Disease Therapies, Keck Graduate Institute, USA
Editorial Board:
Sabina Antoniu, Pulmonary Disease Division, University of Medicine and Pharmacy, Romania
Martin Braddock, Global Medicines Development, AstraZeneca R&D, UK
Maria Luisa Brandi, Unit of Bone and Mineral Metabolism, University of Florence, Italy
Roberto Giugliani, Department of Genetics, Federal University of Rio Grande do Sul, Brazil
Stephen Groft, Director, Office of Rare Diseases, National Institutes of Health, USA
Aaron S. Kesselheim, Harvard Medical School and Brigham and Women's Hospital, USA
Stephen Kingsmore, Center for Pediatric Genomic Medicine, Children's Mercy Hospitals and Clinics, USA
Alex MacKenzie, Pediatrics, CHEO, University of Ottawa, Canada
Maria Mavris, EURORDIS - European Organisation for Rare Diseases, France
Khue Nguyen, University of California, San Diego, USA
Daniel O'Connor, Medicines and Healthcare Products Regulatory Agency, UK
Manuel Posada, Director, Research Institute for Rare Diseases, Instituto de Salud Carlos III, Spain
Peter L. Saltonstall, President and CEO, National Organization for Rare Disorders (NORD), USA
Michael Shy, Division of Neuromuscular Medicine, University of Iowa, USA
Ali Taher, Division of Hematology/Oncology, American University of Beirut Medical Centre, Lebanon
Eleftherios Tsiridis, Academic Department of Trauma and Orthopaedics, University of Leeds, UK