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Jiangxi Province Children's Hospital, First People's Hospital of Hangzhou, Shengjing Hospital, The First Hospital of Jilin University, Chengdu Women's and Children's Central Hospital, Children's Hospital of Chongqing Medical University, Inner Mongolia People's Hospital, Sichuan Provincial People's Hospital, Wuhan Union Hospital, China, Third Affiliated Hospital of Zhengzhou University, Children's Hospital of Soochow University, Yuying Children's Hospital of Wenzhou Medical University, Beijing Children's Hospital, and Qilu Hospital of Shandong University

Food and Drug Administration (FDA), Case Western Reserve University, Grifols Therapeutics LLC, and Deepa Manwani, Director, Sickle Cell Disease Program, Children's Hospital at Montefiore

Takeda and National Cancer Institute (NCI)

Takeda Development Center Americas, Inc.

CSL Behring

National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) and James Andrews, Assistant Professor of Medicine

argenx and Chafic Karam, Staff Physician and Associate Professor of Clinical Neurology

Kanecia Obie Zimmerman, Associate Professor of Pediatrics

Grifols Biologicals, LLC, Dysautonomia International, and Steven Vernino MD PhD, PROFESSOR

Xiao Hui Zhang, Vice President of Peking University Institute of Hematology

Wang Miao, professor

Paul Szabolcs, Chief, Division of Blood and Marrow Transplantation and Cellular Therapy

Andrew M Courtwright, Assistant Professor of Medicine

Takeda Development Center Americas, Inc. and Baxalta Innovations GmbH, now part of Takeda

Kanecia Obie Zimmerman, Associate Professor of Pediatrics

Blood and Marrow Transplant Clinical Trials Network, National Cancer Institute (NCI), National Marrow Donor Program, and National Heart, Lung, and Blood Institute (NHLBI)

National Cancer Institute (NCI)

The First Affiliated Hospital of Xiamen University, Zhangzhou Affiliated Hospital of Fujian Medical University, Quanzhou First Hospital Affiliated to Fujian Medical University, Nanping First Hospital Affiliated to Fujian Medical University, The Second Hospital of Anhui Medical University, The Affiliated Hospital Of Guizhou Medical University, Longyan City First Hospital, and The Affiliated Hospital of Putian University

National Heart, Lung, and Blood Institute (NHLBI)

State Institution, Zaporizhzhia Medical Academy of Post-Graduate Education Ministry of Health of Ukraine and Ivano-Frankivsk National Medical University

David Ziring, Associate Director, Pediatric IBD Center

Thomas Abell, The Arthur M Schoen MD Chair in Gastroenterology

Boston Children's Hospital, University of California, San Francisco, and Amanda Grimes, Assistant Professor

Children's Hospital Los Angeles

Luigi Brunetti, Associate Professor

National Institutes of Health (NIH)

Centers for Disease Control and Prevention

Harry W. Schroeder, Jr., MD PhD, Principal Investigator

National Institute for Health Research, United Kingdom, CSL Behring, University of Liverpool, University College London Hospitals, Guy's and St Thomas' NHS Foundation Trust, Liverpool University Hospitals NHS Foundation Trust, and Great Ormond Street Hospital for Children NHS Foundation Trust

Swedish Orphan Biovitrum

University of Oxford and Alasdair Coles, Revd. Prof. Alasdair Coles, Chief Investigator