Clinical Trials on X-bundet trombocytopeni
Total 3607 results
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Talaris Therapeutics Inc.Duke UniversityTerminatedNiemann-Pick Disease | Alpha-mannosidosis | Tay Sachs Disease | Sandhoff Disease | Metachromatic Leukodystrophy (MLD) | Hurler-Scheie Syndrome | Hurler Syndrome (MPS I) | Hunter Syndrome (MPS II) | Sanfilippo Syndrome (MPS III) | Krabbe Disease (Globoid Leukodystrophy) | Adrenoleukodystrophy (ALD and AMN) | Pelizaeus...United States
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Neuroganics LLCRamos Injury Law FirmRecruitingCOVID-19 | Vaccine Adverse Reaction | Vaccine Reaction | Vasculitis | Neuritis | Thrombocytopenia | Influenza | Corona Virus Infection | Blood Clot | Guillain-Barré | Vaccine or Biological Substance, Unspecified Causing Adverse Effects in Therapeutic Use | GWAS | Genetic SequencingUnited States
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GlaxoSmithKlineTerminatedLiver Diseases | HIV Infection | Thrombocytopenia | Hepatitis C Virus | HBV | NASH - Nonalcoholic Steatohepatitis | Human Immunodeficiency Virus | Chronic Liver Disease | HCV | Non-alcoholic Steatohepatitis | Hepatitis B VirusUnited States, Spain, Taiwan, Italy, Belgium, France, Korea, Republic of, Canada, Russian Federation, Pakistan, Poland, India, Argentina
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University Health Network, TorontoEnrolling by invitationParkinson Disease | Dementia | Alzheimer Disease | Vascular Dementia | Frontotemporal Lobar Degeneration | Neuro-Degenerative Disease | Caregiver BurnoutCanada
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Caroline Michaela KistorpSanofiNot yet recruitingChronic Kidney Diseases | Fabry DiseaseDenmark
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American Thrombosis and Hemostasis NetworkSanofi; Pfizer; Genentech, Inc.; CSL Behring; Hemophilia of Georgia, Inc.; Hemab TherapeuticsRecruitingHemophilia | Thrombosis | Thrombophilia | Sickle Cell Disease | Thalassemia | Bleeding Disorder | Von Willebrand Diseases | Factor IX Deficiency | Connective Tissue Disorder | Factor VIII Deficiency | Hematologic Disorder | Rare Bleeding Disorder | Platelet DisorderUnited States
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Wuerzburg University HospitalTakedaEnrolling by invitationLysosomal Storage Diseases | Fabry Disease | Fabry Disease, Cardiac Variant | HCM - Hypertrophic Cardiomyopathy | Anderson Fabry DiseaseGermany
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CENTOGENE GmbH RostockWithdrawnChronic Kidney Disease | Hearing Loss | Angiokeratomas | Ocular AbnormalitiesSri Lanka, India, Germany
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Melbourne HealthSanofiWithdrawnLysosomal Storage Diseases | Fabry Disease | Niemann-Pick Disease, Type C | Gaucher DiseaseAustralia
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XperiomeThe Touro College and University SystemWithdrawnHemophilia A | Hemophilia B | Idiopathic Pulmonary Fibrosis | Myasthenia Gravis | Cystic Fibrosis | Sickle Cell Disease
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William ShomaliIncyte CorporationRecruitingEosinophilia | Hypereosinophilic Syndrome | Blasts More Than 5 Percent of Bone Marrow Nucleated Cells | Splenomegaly | Hepatomegaly | Chronic Eosinophilic Leukemia, Not Otherwise Specified | JAK2 Gene Mutation | BCR-JAK2 Fusion Protein Expression | Blasts 20 Percent or Less of Peripheral Blood White... and other conditionsUnited States
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Eunice Kennedy Shriver National Institute of Child...RecruitingCHILD Syndrome | Smith Lemli Opitz Syndrome | Lathosterolosis | DesmosterolosisUnited States
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Jerry Vockley, MD, PhDUltragenyx Pharmaceutical IncNo longer availableBarth Syndrome | Mitochondrial Trifunctional Protein Deficiency | Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency | Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2) | Long-chain Hydroxyacyl-CoA Dehydrogenase Deficiency | Glycogen Storage Disorders | Pyruvate Carboxylase Deficiency... and other conditionsUnited States
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Massachusetts General HospitalCompletedFabry Disease | Mitochondrial Disease | Small Fiber Neuropathy | Ehlers Danlos SyndromeUnited States
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Masonic Cancer Center, University of MinnesotaRecruitingHurler Syndrome | Sphingolipidoses | Peroxisomal Disorders | Metachromatic Leukodystrophy | Alpha-Mannosidosis | Hunter Syndrome | Mucopolysaccharidosis Disorders | Maroteaux Lamy Syndrome | Sly Syndrome | Fucosidosis | Aspartylglucosaminuria | Glycoprotein Metabolic Disorders | Recessive Leukodystrophies | Globoid... and other conditionsUnited States
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Institute of Hematology & Blood Diseases HospitalRecruitingLymphoma | Acute Myeloid Leukemia | Leukemia | Hemophilia A | Hemophilia B | Hemophilia | Multiple Myeloma | Myelodysplastic Syndrome | Bone Marrow Transplantation | MDS | Aplastic Anemia | Bleeding Disorder | Myeloma, Multiple | Myelomas, Multiple | Leukemias, Acute Myeloid | Myeloid Leukemias, Acute | Hemophilia As | Hemophilia... and other conditionsChina
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Wuhan UniversityPeking University; PfizerUnknownThe Primary Evaluation is the Change From Baseline at End of Study for CDSS Total Scores. | The Secondary Efficacy Evaluations Include: MADRS, PANSS Total Scores and Subscales and Responder Rate; CGI-S and CGI-I Scales. | Safety Evaluations Include: Laboratory Test Abnormalities; BARS... and other conditions
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Fred Hutchinson Cancer CenterNational Cancer Institute (NCI)TerminatedAcute Myeloid Leukemia | Primary Myelofibrosis | Thrombocytopenia | Acute Lymphoblastic Leukemia | Chronic Lymphocytic Leukemia | Venous Thromboembolism | Myelodysplastic Syndrome | B-Cell Non-Hodgkin Lymphoma | Hematopoietic Cell Transplantation Recipient | Chronic Myelogenous Leukemia, BCR-ABL1 Positive and other conditionsUnited States
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Natera, Inc.Children's Hospital of Philadelphia; Montefiore Medical Center; University of... and other collaboratorsCompletedPrader-Willi Syndrome | DiGeorge Syndrome | 22q11 Deletion Syndrome | Angelman Syndrome | Trisomy 21 | Trisomy 18 | Trisomy 13 | Monosomy X | Sex Chromosome Abnormalities | 1p36 Deletion Syndrome | Cri-du-Chat SyndromeUnited States, Australia, Ireland, Spain, Sweden, United Kingdom
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National Taiwan University HospitalUnknownHypertension | Diabetes | Dementia | Alzheimer Disease | Fabry Disease | Dry Eye Syndrome | Metabolic Disease | Healthy Individuals | Ocular Rosacea | Limbal Insufficiency | Phlyctenulosis | Graft Versus Host Disease in EyeTaiwan
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Baylor College of MedicineThe Methodist Hospital Research Institute; Center for Cell and Gene Therapy...Active, not recruitingAcute Myeloid Leukemia | Acute Lymphoblastic Leukemia | Non Hodgkin Lymphoma | Myelodysplastic Syndrome | Chronic Myelogenous Leukemia | Hemophagocytic Lymphohistiocytosis | Epstein Barr Virus Infection | X-linked Lymphoproliferative Disease | Hemophagocytic Syndrome | Familial Hemophagocytic LymphohistiocytosisUnited States
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Mayo ClinicRecruitingHematologic Neoplasms | Myeloid Malignancy | Cytopenia | Bone Marrow Failure Syndrome | Clonal Cytopenia of Undetermined Significance | Clonal Hematopoiesis of Indeterminate Potential | Low Risk Myelodysplastic Syndrome | Inherited Bone Marrow Failure Syndrome | Clonal Expansion | Hereditary Neoplastic... and other conditionsUnited States
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Drexel UniversityJohns Hopkins University; National Institute on Aging (NIA); University of California...Active, not recruitingDementia | Alzheimer Disease | Frontotemporal Dementia | Vascular Dementia | Dementia With Lewy Bodies | Caregiver Stress Syndrome | Caregiver BurnoutUnited States
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Fred Hutchinson Cancer CenterCompletedMyelodysplastic Syndrome (MDS) | Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive | Chronic Myelogenous Leukemia, BCR-ABL1 Positive | Acute Biphenotypic Leukemia | Acute Myeloid Leukemia in Remission | Acute Lymphoblastic Leukemia in Remission | Blasts Under 5 Percent of Bone Marrow...United States
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dr. Laura C. G. de Graaff-HerderNot yet recruitingCongenital Adrenal Hyperplasia | Hypogonadotropic Hypogonadism | Growth Hormone Deficiency | Turner Syndrome | Addison's Disease | Klinefelter Syndrome | Combined Pituitary Hormone Deficiency | Androgen Insensitivity Syndrome | Thyroid DysgenesisSpain, United Kingdom, Belgium, Netherlands
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University of Colorado, DenverRecruitingHearing Loss | Smith-Lemli-Opitz Syndrome | Cone-Rod DystrophyUnited States
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National Heart, Lung, and Blood Institute (NHLBI)CompletedCystic Fibrosis | Chronic Granulomatous Disease | Primary Ciliary Dyskinesia | Pseudohypoaldosteronism | Nontuberculous Mycobacterial InfectionUnited States
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Paul SzabolcsEnrolling by invitationChronic Granulomatous Disease (CGD) | Wiskott-Aldrich Syndrome | Severe Chronic Neutropenia | Severe Combined Immunodeficiency (SCID) | Immunodeficiency With Predominant T-cell Defect, Unspecified | Hyper IgE Syndromes | Hyper IgM Deficiencies | Mendelian Susceptibility to Mycobacterial Disease | Common...United States
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ResMedClinical Trial Center North Hamburg Germany; CRI-The Clinical Research Institute...TerminatedPulmonary Disease, Chronic Obstructive | Amyotrophic Lateral Sclerosis | Muscular Dystrophies | Duchenne Muscular Dystrophy | Myotonic Dystrophy | Spinal Cord Injury | Obesity Hypoventilation Syndrome | Kyphoscoliosis | Congenital Central Hypoventilation Syndrome | MyopathiesGermany
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National Taiwan University HospitalRecruitingStroke | Magnetic Resonance Imaging | Fabry Disease | Cerebral Small Vessel Diseases | Cadasil | HTRA1-Related Autosomal Dominant Cerebral Angiopathy | COL4A1-Related Brain Small Vessel Disease With Haemorrhage | Next-generation SequencingTaiwan
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National Center for Research Resources (NCRR)University of California, Los AngelesCompletedThrombocytopenia | Inborn Errors of Metabolism | Metachromatic Leukodystrophy | Fanconi's Anemia | Thalassemia Major | Pure Red-Cell AplasiaUnited States
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Alexion Pharmaceuticals, Inc.Not yet recruitingPregnancy | Paroxysmal Nocturnal Hemoglobinuria (PNH) | Atypical Hemolytic Uremic Syndrome (aHUS) | Ultomiris-exposed Pregnant/ Postpartum | Generalized Myasthenia Gravis (gMG) | Neuromyelitis Optica Spectrum Disorder (NMOSD)
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Fred Hutchinson Cancer CenterNational Cancer Institute (NCI)TerminatedAcute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome | Chronic Myelomonocytic Leukemia | Previously Treated Myelodysplastic Syndrome | Adult Acute Myeloid Leukemia in Remission | Adult Acute Lymphoblastic Leukemia in Remission | Refractory Anemia With Excess Blasts | Refractory... and other conditionsUnited States
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O & O Alpan LLCUnknownFabry Disease | Niemann-Pick Disease | Gaucher Disease | Pompe Disease | Lysosomal Storage DisordersUnited States
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LMU KlinikumUniversity of Pisa; German Federal Ministry of Education and Research; European...RecruitingMitochondrial Diseases | MDS | Mitochondrial Myopathies | MELAS Syndrome | MIDD | Kearns-Sayre Syndrome | MERRF Syndrome | Barth Syndrome | Leigh Syndrome | MNGIE | LHON | Pearson Syndrome | NARP Syndrome | Coenzyme Q10 Deficiency | SANDO | SCAE | MIRAS | CPEOAustria, Germany, Italy
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National Cancer Institute (NCI)RecruitingFanconi Anemia | Dyskeratosis Congenita | Diamond Blackfan Anemia | Shwachman Diamond Syndrome | Inherited Bone Marrow Failure Syndrome, Aplastic AnemiaUnited States
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Sanford HealthNational Ataxia Foundation; Beyond Batten Disease Foundation; Pitt Hopkins Research... and other collaboratorsRecruitingMitochondrial Diseases | Retinitis Pigmentosa | Myasthenia Gravis | Eosinophilic Gastroenteritis | Multiple System Atrophy | Leiomyosarcoma | Leukodystrophy | Anal Fistula | Spinocerebellar Ataxia Type 3 | Friedreich Ataxia | Kennedy Disease | Lyme Disease | Hemophagocytic Lymphohistiocytosis | Spinocerebellar Ataxia... and other conditionsUnited States, Australia
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Sidney Kimmel Cancer Center at Thomas Jefferson...Active, not recruitingAcute Myeloid Leukemia | Hodgkin Lymphoma | Chronic Myelomonocytic Leukemia | Myelodysplastic Syndrome | Plasma Cell Myeloma | Aplastic Anemia | Malignant Neoplasm | Myeloproliferative Neoplasm | Indolent Non-Hodgkin Lymphoma | Acute Myeloid Leukemia in Remission | Refractory Anemia | Refractory Anemia With... and other conditionsUnited States
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23andMe, Inc.Active, not recruitingClear Cell Renal Cell Carcinoma | Fallopian Tube Cancer | Solid Tumor | Neuroendocrine Tumors | Epithelial Ovarian Cancer | Primary Peritoneal Carcinoma | Extensive-stage Small-cell Lung Cancer | MSI-H Cancer | Cancer With A High Tumor Mutational BurdenUnited States, Canada
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ECOG-ACRIN Cancer Research GroupNational Cancer Institute (NCI)Active, not recruitingFinancial Burden Assessment in Patients With Stage I-III Colon or Rectal Cancer Undergoing TreatmentStage III Colon Cancer AJCC v8 | Stage III Rectal Cancer AJCC v8 | Stage IIIA Colon Cancer AJCC v8 | Stage IIIA Rectal Cancer AJCC v8 | Stage IIIB Colon Cancer AJCC v8 | Stage IIIB Rectal Cancer AJCC v8 | Stage IIIC Colon Cancer AJCC v8 | Stage IIIC Rectal Cancer AJCC v8 | Stage IIA Rectal Cancer AJCC... and other conditionsUnited States
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RTI InternationalEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsEnrolling by invitationPrimary Hyperoxaluria Type 3 | Diabetes Mellitus | Hemophilia A | Hemophilia B | Hereditary Fructose Intolerance | Cystic Fibrosis | Factor VII Deficiency | Phenylketonurias | Sickle Cell Disease | Dravet Syndrome | Duchenne Muscular Dystrophy | Prader-Willi Syndrome | Fragile X Syndrome | Chronic Granulomatous Disease and other conditionsUnited States
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Erasmus Medical CenterLeiden University Medical Center; Medical Center Haaglanden; Rijnstate Hospital; Maasstadziekenhu... and other collaboratorsRecruitingHIV Infections | Acquired Immunodeficiency Syndrome | Sexually Transmitted Diseases, Viral | Pneumonia | Psoriasis | Weight Loss | Thrombocytopenia | Leukopenia | Dermatitis, Seborrheic | Herpes Zoster | Lymphadenopathy | Uterine Cervical Dysplasia | Primary Health Care | Diarrhea Chronic | Candida Infection | Infectious... and other conditionsNetherlands
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National Human Genome Research Institute (NHGRI)RecruitingMetabolic Disease | Purine-Pyrimidine Metabolism | AICDA, OMIM *605257, Immunodeficiency With Hyper-IgM, Type 2; HIGM2 | UNG, OMIM *191525, Hyper-IgM Syndrome 5 | NT5C3A<TAB>, OMIM *606224, Anemia, Hemolytic, Due to UMPH1 Deficiency | UMPS, OMIM *613891, Orotic Aciduria | DHODH, OMIM *126064,... and other conditionsUnited States
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Emory UniversityChildren's Healthcare of AtlantaTerminatedSickle Cell Disease | Fanconi Anemia | Thalassemia | Severe Congenital Neutropenia | Wiskott-Aldrich Syndrome | Diamond-Blackfan Anemia | Severe Aplastic Anemia | Glanzmann Thrombasthenia | Leukocyte Adhesion Deficiency | Dyskeratosis-congenita | Chediak-Higashi Syndrome | Chronic-granulomatous Disease | Schwachman-Diamond...United States
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West Kazakhstan Medical UniversityRecruitingOrnithine Transcarbamylase Deficiency | Biotinidase Deficiency | Citrullinemia | Glutaric Acidemia Type II | Argininosuccinic Aciduria | Maple Syrup Urine Disease | Primary Carnitine Deficiency | Homocystinuria | Carnitine Palmitoyltransferase II Deficiency | Arginase Deficiency | Very Long-chain Acyl-CoA... and other conditionsKazakhstan
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Emory UniversityCompletedSickle Cell Disease | Fanconi Anemia | Hurler Syndrome | Chronic Granulomatous Disease | Severe Congenital Neutropenia | Hemophagocytic Lymphohistiocytosis | Wiskott-Aldrich Syndrome | Diamond-Blackfan Anemia | Severe Aplastic Anemia | Glanzmann Thrombasthenia | Shwachman-Diamond Syndrome | Thalassemia Major | Leukocyte... and other conditionsUnited States
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University of PittsburghRecruitingLysosomal Storage Diseases | Leukodystrophy | Niemann-Pick Diseases | Batten Disease | Gaucher Disease | Krabbe Disease | Alpha-Mannosidosis | Osteopetrosis | Tay-Sachs Disease | Pelizaeus-Merzbacher Disease | Sandhoff Disease | GM1 Gangliosidoses | MPS I | MPS II | Vanishing White Matter Disease | Multiple Sulfatase Deficiency... and other conditionsUnited States
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National Cancer Institute (NCI)CompletedThrombocytopenia | Chronic Myelomonocytic Leukemia | Recurrent Adult Acute Myeloid Leukemia | Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities | Adult Acute Myeloid Leukemia With Del(5q) | Adult Acute Myeloid Leukemia With Inv(16)(p13;q22) | Adult Acute Myeloid Leukemia With t(16;16)(p13... and other conditionsUnited States
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Roswell Park Cancer InstituteCompletedPrimary Myelofibrosis | Polycythemia Vera | Chronic Myelomonocytic Leukemia | Recurrent Adult Acute Myeloid Leukemia | Juvenile Myelomonocytic Leukemia | Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue | Nodal Marginal Zone B-cell Lymphoma | Recurrent Adult Burkitt Lymphoma and other conditionsUnited States
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Centre Hospitalier Universitaire de LiegeSanofi; Takeda; University of Liege; Orchard Therapeutics; Centre Hospitalier Régional... and other collaboratorsRecruitingCongenital Adrenal Hyperplasia | Hemophilia A | Hemophilia B | Mucopolysaccharidosis I | Mucopolysaccharidosis II | Cystic Fibrosis | Alpha 1-Antitrypsin Deficiency | Sickle Cell Disease | Fanconi Anemia | Chronic Granulomatous Disease | Wilson Disease | Severe Congenital Neutropenia | Ornithine Transcarbamylase... and other conditionsBelgium