Inherited Blood Disorders (Approved)
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Inherited Blood Disorders (Approved)
Acute Myelofibrosis
Agnogenic Myeloid Metaplasia
Aplastic Anemia
Beta Thalassemia Major
Blackfan-Diamond Anemia
Congenital Amegakaryocytic Thrombocytopenia
Congenital Cytopenia
Congenital Dyserythropoietic Anemia
Dyskeratosis Congenita
Essential Thrombocythemia
Fanconi Anemia
Glanzmann’s Thrombasthenia
Myelodysplastic Syndrome
Paroxysmal Nocturnal Hemoglobinuria
Polycythemia Vera
Pure Red Cell Aplasia
Refractory Anemia with Excess Blasts
Refractory Anemia with Excess Blasts in Transition
Refractory Anemia with Ringed Sideroblasts
Shwachman-Diamond Syndrome
Sickle Cell Disease
Inherited Immune Disorders (Approved)
Adenosine Deaminase Deficiency
Bare Lymphocyte Syndrome
Chediak-Higashi Syndrome
Chronic Granulomatous Disease
Congenital Neutropenia
DiGeorge Syndrome
Evans Syndrome
Fucosidosis
Hemophagocytic Lymphohistiocytosis
Hemophagocytosis Langerhans’ Cell
Histiocytosis X
IKK Gamma Deficiency
Immune Dysregulation,
Polyendocrinopathy, Enteropathy, X-linked
Syndrome Kostmann Syndrome
Myelokathexis
Omenn Syndrome
Phosphorylase Deficiency
Purine Nucleoside
Reticular Dysgenesis
Severe Combined Immunodeficiency
Diseases
Thymic Dysplasia
Wiskott-Aldrich Syndrome
X-linked Agammaglobulinemia
X-Linked Hyper IgM Syndrome
X-Linked Lymphoproliferative Disorder
Inherited Errors in Metabolism (Approved)
Congenital Erythropoietic Porphyria
Agnogenic Myeloid Metaplasia
Gaucher Disease
Hunter Syndrome
Hurler Syndrome
Krabbe Disease
Lesch-Nyhan Syndrome
Mannosidosis
Maroteaux-Lamy Syndrome
Metachromatic Leukodystrophy
Mucolipidosis II
Neuronal Ceroid Lipofuscinosis
Niemann-Pick Disease
Sandhoff Disease
Sanfilippo Syndrome
Scheie Syndrome Sly Syndrome
Tay Sachs
Wolman Disease
X-Linked Adrenoleukodystrophy
Blood Cancers (Approved)
Acute Biphenotypic Leukemia
Acute Lymphocytic Leukemia
Acute Myelogenous Leukemia
Acute Undifferentiated Leukemia
Adult T Cell Leukemia/Lymphoma
Chronic Active Epstein Barr
Chronic Lymphocytic Leukemia
Chronic Myelogenous Leukemia
Chronic Myelomonocytic Leukemia
Ewing Sarcoma
Hodgkin’s Lymphoma
Juvenile Chronic Myelogenous Leukemia
Juvenile Myelomonocytic Leukemia
Myeloid/Natural Killer Cell
PrecursorAcute Leukemia
Non-Hodgkin’s Lymphoma
Prolymphocytic Leukemia
Plasma Cell Leukemia
Leukocyte Adhesion Deficiency
Multiple Myeloma
Neuroblastoma
Rhabdomyosarcoma
Thymoma
Waldenstrom’s Macroglobulinemia
Wilms Tumor
Conditions Under Clinical Trial
Autism
Cerebral Palsy
Spinal Cord Injury
Parkinson's Disease
Lupus
Crohn's Disease
Rheumatoid Arthiritis
Diabetes Type 1 & 2
Lysosomal Storage Disease
HIV
Osteoarthritis
Peripheral Arterial Disease
Alzheimer's disease
Encephalopathy
Global Developmental Delay
Hearing Loss (acquired sensorineural)
Intraventricular Hemorrhage
Stroke
Traumatic Brain Injury
Alopecia Areata
Amyotrophic Lateral Sclerosis
Eczema (Atopic Dermatitis)
Graft-versus-Host Disease
Systemic Sclerosis
Critical Limb Ischemia
Aging Frailty
Epidermolysis Bullosa
Severe Combined Immunodeficiency
Tay-Sachs Disease
Cartilage Injury
Osteochondral Lesion
BronchoPulmonary Dysplasia
Eye Diseases
Liver Failure
Premature Ovarian Failure