TREATABLE DISEASES

• 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)

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• 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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