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TREATABLE DISEASES

APPROVED DISORDERS THAT COULD BE ADDRESSED USING STEM CELLS

  • Leukaemia’s: Leukaemia is a cancer of the blood immune system, cells are called leukocytes or white cells.

    • Acute Lymphoblastic Leukaemia (ALL)

    • Acute Myelogenous Leukaemia (AML)

    • Acute Biphenotypic Leukaemia

    • Acute Undifferentiated Leukaemia

    • Chronic Lymphocytic Leukaemia (CLL)

    • Chronic Myelogenous Leukaemia (CML)

    • Juvenile Chronic Myelogenous Leukaemia (JCML)

    • Juvenile Myelomonocytic Leukaemia (JMML)

 

  • Myelodysplastic Syndromes: Myelodysplastic Syndromes are also called pre-leukaemia.

    • Refractory Anaemia

    • Refractory Anaemia with Ringed Sideroblasts (Sideroblastic anemia)

    • Refractory Anaemia with Excess Blasts

    • Refractory Anaemia with Excess Blasts in Transformation

    • Chronic Myelomonocytic Leukaemia (CMML)

  • Lymphoma: Lymphoma is a cancer of the leukocytes that circulate in the blood and lymph vessels.

    • Hodgkin's Lymphoma

    • Non-Hodgkin's Lymphoma (Burkik's Lymphoma)

 

  • Transplants for Inherited Disorders: Transplants for Inherited Disorders of the Immune System and Other Organs.

    • Cartilage-Hair Hypoplasia

    • Erythropoitic Porphyria

    • Hermansky-Pudlak Syndrome

    • Pearson's Syndrome

    • Shwachman-Diamond Syndrome

    • Systemic Mastocytosis

 

  • Transplants for Inherited Metabolic Disorders: Mucopolysaccharidosis (MPS) Storage Diseases.

    • Hurler Syndrome (MPS-IH)

    • Scheie Syndrome (MPS-IS)

    • Hunter Syndrome (MPS-II)

    • Sanfilippo Syndrome (MPS-III)

    • Morquio Syndrome (MPS-IV)

    • Maroteaux-Lamy Syndrome (MPS-VI)

    • Sly Syndrome (MPS-VII) (beta-glucuronidase deficiency)

    • Mucolipidosis II (I-cell Disease)

 

  • Leukodystrophy Disorders

    • Adrenoleukodystrophy (ALD)

    • Krabbe Disease (Globoid Cell Leukodystrophy)

    • Metachromatic Leukodystrophy

    • Pelizaeus-Merzbacher Disease

 

  • Lysosomal Storage Diseases

    • Niemann-Pick Disease

    • Sandhoff Disease

    • Wolman Disease

 

  • Other Inherited Diseases

    • Lesch-Nyhan Syndrome

    • Osteopetrosis

 

  • Other Disorders of Blood Cell Proliferation: Anaemias are deficiencies or malformations of red cells.

    • Aplastic Anaemia

    • Fanconi Anaemia

    • Congenital Dyserythropoietic Anaemia

    • Paroxysmal Nocturnal Hemoglobinuria (PNH)

 

  • Inherited Red Cell Abnormalities: Red cells contain haemoglobin and carry oxygen to the body.

    • Sickle Cell Disease

    • Beta Thalassemia Major (aka Cooley's Anaemia)

    • Diamond-Blackfan Anaemia

    • Pure Red Cell Aplasia

 

  • Inherited Platelet Abnormalities: Platelets are blood cells needed for clotting.

    • Amegakaryocytosis / Congenital Thrombocytopenia

    • Glanzmann Thrombasthenia

 

  • Inherited Immune System Disorders: Severe Combined Immunodeficiency.

    • SCID with Adenosine Deaminase Deficiency (ADA-SCID)

    • SCID which is X-linked

    • SCID with absence of T & B Cells

    • SCID with absence of T Cells, Normal B Cells

    • Omenn Syndrome

 

  • Inherited Immune System Disorders: Neutropenia’s

    • Infantile Genetic Agranulocytosis (Kostmann Syndrome)

    • Myelokathexis

 

  • Inherited Immune System Disorders: Other

    • Ataxia-Telangiectasia

    • Bare Lymphocyte Syndrome

    • Common Variable Immunodeficiency

    • DiGeorge Syndrome

    • Haemophagocytic Lymphohistiocytosis

    • Leukocyte Adhesion Deficiency

    • Lymphoproliferative Disorders

    • Lymphoproliferative Disorder, X-linked (Susceptibility to Epstein-Barr virus)

    • Wiskok-Aldrich Syndrome

 

  • Myeloproliferative Disorders

    • Acute Myelofibrosis

    • Agnogenic Myeloid Metaplasia (Myelofibrosis)

    • Polycythemia Vera

    • Essential Thrombocythemia

 

  • Phagocyte Disorders: These are immune system cells that engulf and kill foreign organisms.

    • Chediak-Higashi Syndrome Chronic

    • Granulomatous Disease

    • Neutrophil Actin Deficiency

    • Reticular Dysgenesis

 

  • Bone Marrow Cancers

    • Multiple Myeloma

    • Plasma Cell Leukaemia

    • Waldenstrom's Macroglobulinemia Agnogenis Myeloid Metaplasia (Myelofibrosis)

 

  • Solid Tumours: Solid tumours not originating in the blood or immune system.

    • Neuroblastoma

    • Medulloblastoma

    • Retinoblastoma

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