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Centre Hospitalier Régional de la Citadelle, University of Liege, Sanofi, Orchard Therapeutics, Takeda, Zentech-Lacar Company, Leon Fredericq Foundation, and Laurent Servais, Professor

Incyte Corporation, North American Consortium for Histiocytosis, and Cures Within Reach

National Ataxia Foundation, International WAGR Syndrome Association, 4p- Support Group, ML4 Foundation, Cornelia de Lange Syndrome Foundation, Stickler Involved People, Kawasaki Disease Foundation, Klippel-Feil Syndrome Alliance, Klippel-Feil Syndrome Freedom, Hyperacusis Research Limited, Hypersomnia Foundation, Kabuki Syndrome Network, Kleine-Levin Syndrome Foundation, Leiomyosarcoma Direct Research Foundation, Marinesco-Sjogren Syndrome Support Group - NORD, Mucolipidosis Type IV (ML4) Foundation, People with Narcolepsy 4 People with Narcolepsy (PWN4PWN), Soft Bones Incorporated, American Multiple Endocrine Neoplasia Support, Atypical Hemolytic Uremic Syndrome Foundation, All Things Kabuki, Wiedemann-Steiner Syndrome Foundation, Breast Implant Victim Advocates, PROS Foundation, American Behcet's Disease Association, Alstrom United Kingdom, Athymia, Curing Retinal Blindness Foundation, HSAN1E Society, 1p36 Deletion Support and Awareness, The Alagille Syndrome Alliance, Autoinflammatory Alliance, Beyond Batten Disease Foundation, Bohring-Opitz Syndrome Foundation, INC, Cockayne Syndrome Network (Share and Care), CRMO Foundation, Cure VCP Disease,INC, FOD Support, Cystinosis Research Foundation, Global DARE Foundation, Hypnic Jerk-Sleep Myoclonus Support Group, Jansen's Foundation, KCNMA1 Channelopathy International Advocacy Foundation, Kawasaki Disease Foundation Australia, Life with LEMS Foundation, Lowe Syndrome Association, The Malan Syndrome Foundation, Maple Syrup Urine Disease Family Support Group, International Association for Muscle Glycogen Storage Disease (IamGSD), Myhre Syndrome Foundation, DNM1 Families, Nicolaides Baraitser Syndrome (NCBRS) Worldwide Foundation, The PBCers Organization, Pitt Hopkins Research Foundation, Recurrent Meningitis Association, Recurrent Respiratory Papillomatosis Foundation, Remember the Girls, Smith-Kingsmore Syndrome Foundation, SPG Research Foundation, Team Telomere, Transient Global Amnesia Project, The Charlotte & Gwenyth Gray Foundation, The Cute Syndrome Foundation, The Maddi Foundation, White Sutton Syndrome Foundation, Zmynd11 Gene Disorder, Cauda Equina Foundation, Inc, Tango2 Research Foundation, Noah's Hope - Hope4Bridget Foundation, Project Sebastian, SMC1A Epilepsy Foundation, International Foundation for Gastrointestinal Disorders, Endosalpingiosis Foundation, Inc, International Sacral Agenesis/Caudal Regression Association (ISACRA), Scheuermann's Disease Fund, Batten Disease Support and Research Association, Kennedy's Disease Association, Cure Mito Foundation, Warburg Micro Research Foundation, Cure Mucolipidosis, Riaan Research Initiative, CureARS A NJ Nonprofit Corporation, CACNA1H Alliance, IMBS Alliance, SHINE-Syndrome Foundaion, Non- Ketotic Hyperglycinemia (NKH) Crusaders, Hypertrophic Olivary Degeneration Association (HODA), National Organization for Disorders of the Corpus Callosum (NODCC), Team4Travis, Taylor's Tale Foundation, Lambert Eaton (LEMS) Family Association, BARE Inc, STAG1 Gene Foundation, Coffin Lowry Syndrome Foundation, BLFS Incorporate, Aniridia North America, Cure Blau Syndrome Foundation, ARG1D Foundation, CURE HSPB8 Myopathy, International Society of Mannosidosis and Related Disorders, TBX4Life, Cure DHDDS, MANDKind Foundation, Krishnan Family Foundation, and SPATA Foundation

Center for Cell and Gene Therapy, Baylor College of Medicine, The Methodist Hospital Research Institute, and Erin Morales, Assistant Professor

Sobi, Inc. and Baylor College of Medicine

Incyte Corporation and Aaron Logan, MD, Principal Investigator

Daihong Liu, Director

Zhao Wang, Professor and Head of hematology

Aarhus University Hospital

Light Chain Bioscience - Novimmune SA

Brigham and Women's Hospital and Harvard Medical School (HMS and HSDM)

Live Like Bella Pediatric Cancer Research

Zhao Wang, director of department

shifeng Lou, Director

Zhao Wang, Professor and Head of hematology

The Methodist Hospital Research Institute, Center for Cell and Gene Therapy, Baylor College of Medicine, and Malcolm Brenner, Director/Professor, Center for Cell and Gene Therapy

Gaurav Goyal, Principal Investigator

Children's Hospital Medical Center, Cincinnati, Rabin Medical Center, Sheba Medical Center, and Schneider Children's Medical Center, Israel

Prof. Franca Fagioli, Clinical Professor

Zhao Wang, Clinical Professor

Zhao Wang, Professor and Head of hematology

Seventh Framework Programme

Zhao Wang, Head of the hematology department

Claudia Spies, Head of the Department of Anesthesiology and Operative Intensive Care Medicine (CCM, CVK), Charité - Universitätsmedizin Berlin

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

Gunnar Lachmann, Specialist in Anesthesiology of the Department of Anesthesiology and Intensive Care Medicine CVK/CCM

Zhao Wang, Professor and Head of hematology

The Second Hospital of Anhui Medical University, Union hospital of Fujian Medical University, Children's Hospital of Fudan University, Zunyi Medical College, Hunan Provincial People's Hospital, Tongji Hospital, Children's Hospital of Nanjing Medical University, The Affiliated Hospital of Qingdao University, Qilu Hospital of Shandong University, Shanghai Children's Medical Center, Xinhua Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai Children's Hospital, Shenzhen Children's Hospital, West China Second University Hospital, Children's Hospital of Soochow University, Second Affiliated Hospital of Wenzhou Medical University, Wuhan Children's Hospital, Institute of Hematology & Blood Diseases Hospital, China, The Third Xiangya Hospital of Central South University, First Affiliated Hospital, Sun Yat-Sen University, and Xiaojun Xu, chief physician

Zhao Wang, Professor and Head of hematology

Seventh Framework Programme

Zhao Wang, Clinical Professor