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Diseases Treated with Cord Blood

Today there are more than 80 diseases and conditions being treated with cord blood stem cells and the list continues to grow.

Blood Disorders
Acute Myelofibrosis 
Agnogenic Myeloid Metaplasia (Myelofibrosis)
Aplastic Anemia (Severe)
Beta Thalassemia Major
Blackfan-Diamond Anemia
Congenital Amegakaryocytic Thrombocytopenia (CAT)
Congenital Cytopenia
Congenital Dyserythropoietic Anemia
Dyskeratosis Congenita
Essential Thrombocythemia
Fanconi Anemia

Glanzmann’s Thrombasthenia
Myelodysplastic Syndrome
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Polycythemia Vera
Pure Red Cell Aplasia
Refractory Anemia with Excess Blasts (RAEB)
Refractory Anemia with Excess Blasts in Transition (RAEB-T)
Refractory Anemia with Ringed Sideroblasts (RARS)
Shwachman-Diamond Syndrome
Sickle Cell Disease


Acute Biphenotypic Leukemia
Acute Lymphocytic Leukemia (ALL)
Acute Myelogenous Leukemia (AML)
Acute Undifferentiated Leukemia
Adult T Cell Leukemia/Lymphoma
Chronic Active Epstein Barr
Chronic Lymphocytic Leukemia (CLL)
Chronic Myelogenous Leukemia (CML)
Chronic Myelomonocytic Leukemia (CMML)
Ewing Sarcoma
Hodgkin’s Lymphoma
Juvenile Chronic Myelogenous Leukemia (JCML)
Juvenile Myelomonocytic Leukemia (JMML)​

Myeloid/Natural Killer (NK) Cell PrecursorAcute Leukemia
Non-Hodgkin’s Lymphoma
Prolymphocytic Leukemia
Plasma Cell Leukemia
Leukocyte Adhesion Deficiency
Multiple Myeloma
Thymoma (Thymic Carcinoma)
Waldenstrom’s Macroglobulinemia
Wilms Tumor


Immune Disorders
Adenosine Deaminase Deficiency (SCID)
Bare Lymphocyte Syndrome (SCID)
Chediak-Higashi Syndrome (SCID)
Chronic Granulomatous Disease
Congenital Neutropenia
DiGeorge Syndrome
Evans Syndrome
Hemophagocytic Lymphohistiocytosis (HLH)
Hemophagocytosis Langerhans’ Cell Histiocytosis (Histiocytosis X)
IKK Gamma Deficiency (NEMO Deficiency)
Immune Dysregulation, Polyendocrinopathy, Enteropathy, X-linked (IPEX) Syndrome

Kostmann Syndrome (SCID)
Omenn Syndrome (SCID)
Phosphorylase Deficiency (SCID)
Purine Nucleoside (SCID)
Reticular Dysgenesis (SCID)
Severe Combined Immunodeficiency Diseases (SCID)
Thymic Dysplasia
Wiskott-Aldrich Syndrome
X-linked Agammaglobulinemia
X-Linked Hyper IgM Syndrome
X-Linked Lymphoproliferative Disorder


Metabolic Disorders
Congenital Erythropoietic Porphyria (Gunther Disease)
Gaucher Disease
Hunter Syndrome (MPS-II)
Hurler Syndrome (MPS-IH)
Krabbe Disease
Lesch-Nyhan Syndrome
Maroteaux-Lamy Syndrome (MPS-VI)
Metachromatic Leukodystrophy
Mucolipidosis II (I-cell Disease)

Neuronal Ceroid Lipofuscinosis (Batten Disease)
Niemann-Pick Disease
Sandhoff Disease
Sanfilippo Syndrome (MPS-III)
Scheie Syndrome (MPS-IS)
Sly Syndrome (MPS-VII)
Tay Sachs
Wolman Disease
X-Linked Adrenoleukodystrophy


Note: Some diseases may be known by other names. The use of umbilical cord blood stem cells to treat many diseases and conditions is typically considered standard therapy, while the treatment for other conditions may still be in a clinical trial phase. Currently, stem cells are being used in trials for the treatment of brain injuries, cerebral palsy, autism, diabetes, spinal cord injuries, heart disease, liver disease, and others. These conditions have not been included in this list.

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