Enrollment
Families were recruited in Lithuania, the United Kingdom, France, Saudi Arabia, the USA, and Singapore. The institutional review boards of the Vilnius University Faculty of Medicine, NHS Foundation Trust, Hôpitaux Universitaires de Strasbourg, King Faisal Specialist Hospital and Research Center, the Children’s Hospital of Philadelphia, “Hospices Civils de Lyon,” and KK Women’s and Children’s Hospital approved this study. Participants were enrolled after written informed consent was obtained from parents or legal guardians. The clinical evaluation included medical history interviews, a physical examination, medical imaging as appropriate, and review of medical records.