Potentially Treatable Diseases With Stem Cell

Acute Leukaemias

Acute Biphenotypic Leukaemia. Acute Lymphocytic Leukaemia (ALL). Acute Myelogenous Leukaemia (AML).
Acute Lymphoblastic Leukaemia. Acute Undifferentiated Leukaemia

Chronic Leukaemias

Chronic Lymphocytic Leukaemia (CLL). Chronic Myelogenous Leukaemia (CML). Juvenile Chronic Myelogenous
Leukaemia (JCML). Juvenile Myelomonocytic Leukaemia (JMML). Juvenile Myelogenous Leukaemia

Myelodysplastic Syndromes

Amyloidosis. Chronic Myelomonocytic Leukaemia (CMML). Refractory Anaemia (RA). Refractory Anaemia with Excess Blasts (RAEB). Refractory Anaemia with Excess Blasts in Transformation (RAEB-T). Refractory Anaemia with Ringed Sideroblasts (RARS)

Myeloproliferative Disorders

Acute Myelofibrosis. Agnogenic Myeloid Metaplasia (Myelofibrosis). Essential Thrombocythemia. Polycythemia Vera

Lymphoproliferative Disorders

Hodgkin’s Disease. Non-Hodgkin’s Lymphoma. Prolymphocytic Leukaemia

Inherited Metabolic Disorders

Adrenoleukodystrophy. Alpha Mannosidosis. Beta-Glucuronidase Deficiency (MPS-Vll). Gaucher’s Disease. Hunter’s Syndrome (MPS-ll). Hurler’s Syndrome (MPS-lH). Krabbe Disease. Maroteaux-Lamy Syndrome (MPS-Vl). Metachromatic Leukodystrophy. Morquio Syndrome (MPS-lV). Mucolipidosis II (l-cell Disease). Mucopolysaccharidoses (MPS). Niemann-Pick Disease. Sanfilippo Syndrome (MPS-lll). Scheie Syndrome (MPS-lS). Sly Syndrome. Wolman Disease

Inherited Platelet Abnormalities

Amegakaryocytosis / Congenital Thrombocytopenia

Inherited Erythrocyte Abnormalities

Beta Thalassaemia Major. Blackfan-Diamond Anaemia. Pure Red Cell Aplasia. Sickle Cell Disease

Phagocyte Disorders

Chediak-Higashi Syndrome. Chronic Granulomatous Disease. Neutrophil Actin Deficiency. Reticular Dysgenesis

Histiocytic Disorders

Familial Erythrophagocytic. Lymphohistiocytosis. Hemophagocytosis. Histiocytosis-X. Langerhans’ Cell Histiocytosis

Congenital (Inherited) Immune System Disorders

Absence of T & B Cells SCID. Absence of T Cells, Normal B Cell SCID. Ataxia-Telangiectasia. Bare Lymphocyte Syndrome. Common Variable Immunodeficiency. DiGeorge Syndrome. Kostmann Syndrome. Leukocyte Adhesion Deficiency. Omenn’s Syndrome. Severe Combined Immunodeficiency (SCID). SCID with Adenosine Deaminase. Deficiency. Wiskott-Aldrich Syndrome. X-Linked Lymphoproliferative Disorder

Stem Cell Disorders

Aplastic Anaemia (Severe). Congenital Cytopenia. Dyskeratosis Congenita. Fanconi Anaemia.
Paroxysmal Nocturnal Hemoglobinuria (PNH)

Plasma Cell Disorders

Multiple Myeloma. Plasma Cell Leukaemia. Waldenstrom’s Macroglobulinemia

Other Inherited Disorders

Cartilage-Hair Hypoplasia. Ceroid Lipofuscinosis. Congenital Erythropoietic Porphyria. Glanzmann Thrombasthenia
Lesch-Nyhan Syndrome. Osteopetrosis. Tay Sachs Disease

Emerging Treatments

Diabetes. Cerebral Palsy. Brain Injury. Heart Diseases. Stroke