Clinical Trials

Cytopenia, Anemia, Refractory, with Excess of Blasts, Fanconi Anemia, Myelodysplastic Syndromes, Leukemia, Myeloid, Acute

Ataxia with vitamin E deficiency, Beta ketothiolase deficiency, Carnitine palmitoyl transferase 1A deficiency, Carnitine palmitoyl transferase 2 deficiency, dopamine beta hydroxylase deficiency, Malonic aciduria, Nephrosis, congenital, Cholestasis, progressive familial intrahepatic 1, Medium chain acyl CoA dehydrogenase deficiency, Pyridoxine-dependent epilepsy, Neonatal-onset citrullinemia type 2, Systemic carnitine deficiency, Glut1 Deficiency Syndrome, Glutaric Acidemia I, Timothy syndrome, Brown-Vialetto-Van Laere syndrome, Methylmalonic acidemia, Aromatic amino acid decarboxylase deficiency, Succinyl-CoA:3-oxoacid CoA transferase deficiency, Segawa syndrome, autosomal recessive, Neutropenia, Severe Congenital, Autosomal Recessive 3, Basal ganglia disease, biotin-responsive, Sucrase-isomaltase deficiency, congenital, Acidemia, isovaleric, 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency, HHH syndrome, Glucose-Galactose Malabsorption, Hypophosphatasia, Infantile, Dystonia, Dopa-Responsive, due to Sepiapterin Reductase Deficiency, Carnitine-Acylcarnitine Translocase Deficiency, Hypermethioninemia, Cardiomyopathy, Familial Hypertrophic, 4, Pyridoxamine 5-Prime-Phosphate Oxidase Deficiency, Phosphoglycerate Dehydrogenase Deficiency, Trifunctional Protein Deficiency With Myopathy And Neuropathy, Acyl-CoA Dehydrogenase Family, Member 9, Deficiency of, Phosphoserine Aminotransferase Deficiency, Inflammatory Bowel Disease 25, Autosomal Recessive, 3-Hydroxy-3-Methylglutaryl-CoA Synthase 2 Deficiency, Neurodegeneration Due To Cerebral Folate Transport Deficiency, Glycogen Storage Disease XIV, Combined Pituitary Hormone Deficiency, Shwachman-Diamond Syndrome, Polymorphic Catecholaminergic Ventricular Tachycardia, Adrenal Hyperplasia, Congenital, Adrenoleukodystrophy, Anemia, Sickle Cell, Charcot-Marie-Tooth Disease, Chediak-Higashi Syndrome, Hemophilia B, Congenital Hypothyroidism, Crigler-Najjar Syndrome, Cystic Fibrosis, Cystinosis, Diabetes Mellitus, Type 2, Fanconi Syndrome, Fanconi Anemia, Fructose Intolerance, Galactosemias, Gaucher Disease, Glucosephosphate Dehydrogenase Deficiency, Glycogen Storage Disease, Glycogen Storage Disease Type II, Granulomatous Disease, Chronic, Hemophilia A, Hepatolenticular Degeneration, Homocystinuria, Hyperoxaluria, Primary, Menkes Kinky Hair Syndrome, Leukodystrophy, Metachromatic, Mucopolysaccharidosis I, Hyperlipoproteinemia Type I, Maple Syrup Urine Disease, Mucopolysaccharidosis IV, Mucopolysaccharidosis VI, Nephritis, Hereditary, Neuronal Ceroid-Lipofuscinoses, Phenylketonurias, Pseudohypoaldosteronism, Retinoblastoma, Riboflavin Deficiency, Vitamin B 12 Deficiency, Wiskott-Aldrich Syndrome, Wolman Disease, Fructose-1,6-Diphosphatase Deficiency, Severe Combined Immunodeficiency, Mucopolysaccharidosis II, Mucopolysaccharidosis VII, Stiff-Person Syndrome, alpha-Thalassemia, Diabetes Insipidus, Nephrogenic, Smith-Lemli-Opitz Syndrome, alpha 1-Antitrypsin Deficiency, Hyperglycinemia, Nonketotic, Citrullinemia, Hyperargininemia, Ornithine Carbamoyltransferase Deficiency Disease, Carbamoyl-Phosphate Synthase I Deficiency Disease, Tyrosinemias, Myasthenic Syndromes, Congenital, Biotinidase Deficiency, Holocarboxylase Synthetase Deficiency, Anemia, Diamond-Blackfan, Jervell-Lange Nielsen Syndrome, Congenital Hyperinsulinism, Andersen Syndrome, Lymphohistiocytosis, Hemophagocytic, Familial Hypophosphatemic Rickets, Aspartylglucosaminuria, Propionic Acidemia, Argininosuccinic Aciduria

Primary Immunodeficiency Diseases, Cytopenia, Anemia, Anemia, Aplastic, Anemia, Sickle Cell, Fanconi Anemia, Hemoglobinopathies, Leukemia, Lymphoma, Non-Hodgkin, Myelodysplastic Syndromes, Thalassemia, Leukemia, Myeloid, Acute, Hematologic Neoplasms, Anemia, Diamond-Blackfan, Precursor Cell Lymphoblastic Leukemia-Lymphoma

Fanconi Anemia, Multiple Myeloma, Myeloproliferative Disorders, Neoplasms, Plasma Cell, Myelodysplastic-Myeloproliferative Diseases

Fanconi Anemia, Myelodysplastic Syndromes, Leukemia, Myeloid, Acute

Pdgfra-Associated Chronic Eosinophilic Leukemia, Congenital Abnormalities, Anemia, Aplastic, Burkitt Lymphoma, Fanconi Anemia, Hemoglobinuria, Paroxysmal, Hodgkin Disease, Immunoblastic Lymphadenopathy, Lymphoma, Follicular, Lymphoma, Non-Hodgkin, Waldenstrom Macroglobulinemia, Multiple Myeloma, Mycosis Fungoides, Myeloproliferative Disorders, Recurrence, Sezary Syndrome, Leukemia, Lymphocytic, Chronic, B-Cell, Leukemia, Myeloid, Accelerated Phase, Leukemia, Myeloid, Chronic-Phase, Leukemia, Neutrophilic, Chronic, Leukemia, Myeloid, Acute, Leukemia, Myelomonocytic, Chronic, Lymphoma, Large-Cell, Immunoblastic, Lymphoma, Large B-Cell, Diffuse, Lymphoma, T-Cell, Cutaneous, Lymphoma, Large-Cell, Anaplastic, Lymphoma, B-Cell, Marginal Zone, Lymphoma, Mantle-Cell, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Precursor T-Cell Lymphoblastic Leukemia-Lymphoma, Lymphoma, Extranodal NK-T-Cell, Leukemia, Myelomonocytic, Juvenile, Leukemia, Myeloid, Chronic, Atypical, BCR-ABL Negative, Dendritic Cell Sarcoma, Interdigitating, Primary Myelofibrosis

Pdgfra-Associated Chronic Eosinophilic Leukemia, Congenital Abnormalities, Blast Crisis, Burkitt Lymphoma, Fanconi Anemia, Graft vs Host Disease, Hodgkin Disease, Leukemia, Leukemia, Hairy Cell, Lymphoma, Lymphoma, Follicular, Lymphoma, Non-Hodgkin, Multiple Myeloma, Mycosis Fungoides, Myelodysplastic Syndromes, Myeloproliferative Disorders, Recurrence, Sezary Syndrome, Leukemia, Lymphocytic, Chronic, B-Cell, Leukemia, Myeloid, Accelerated Phase, Leukemia, Myeloid, Chronic-Phase, Leukemia, Neutrophilic, Chronic, Leukemia, Myeloid, Acute, Leukemia, Myelomonocytic, Chronic, Lymphoma, Large-Cell, Immunoblastic, Lymphoma, Large B-Cell, Diffuse, Lymphoma, T-Cell, Cutaneous, Lymphoma, B-Cell, Marginal Zone, Lymphoma, Mantle-Cell, Anemia, Diamond-Blackfan, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Neoplasms, Plasma Cell, Leukemia, Myelomonocytic, Juvenile, Myelodysplastic-Myeloproliferative Diseases, Leukemia, Myeloid, Chronic, Atypical, BCR-ABL Negative, Dendritic Cell Sarcoma, Interdigitating, Primary Myelofibrosis

Burkitt Lymphoma, Fanconi Anemia, Leukemia, Lymphoma, Myelodysplastic Syndromes, Neuroblastoma, Recurrence, Sarcoma, Leukemia, Myeloid, Chronic-Phase, Neuroectodermal Tumors, Primitive, Peripheral, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Myelomonocytic, Juvenile, Dendritic Cell Sarcoma, Interdigitating

Anemia, Aplastic, Fanconi Anemia, Hematologic Diseases, Leukemia, Myelodysplastic Syndromes, Leukemia, Biphenotypic, Acute, Leukemia, Myeloid, Acute, Hematologic Neoplasms, Rare Diseases, Precursor Cell Lymphoblastic Leukemia-Lymphoma