1. La Rabta Hospital; Tunis; Tunisia
2. Sanatorio Español; Asunción; Paraguay
3. Shire Human Genetic Therapies, Inc.; Lexington; Massachusetts
4. Shaare Zedek Medical Center and Hebrew University-Hadassah Medical School; Jerusalem; Israel
5. All India Institute of Medical Sciences; New Delhi; India
6. Hematology Research Center; Moscow; Russia
7. Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER) and Hospital Universitario Miguel Servet; Zaragoza; Spain
8. Your Health S. A.; Buenos Aires; Argentina
9. KEM Hospital Research Centre; Pune; India
10. Duke University Medical Center; Durham; North Carolina
11. Malabar Institute of Medical Sciences Ltd.; Calicut; India
12. Royal Free Hospital, University College London School of Medicine; London; United Kingdom