Gene/Protein Disease Symptom Drug Enzyme Compound
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Query: UMLS:C0027819 (neuroblastoma)
 27,800 document(s) hit in 31,850,051 MEDLINE articles (0.00 seconds)

The first meeting devoted specifically to BMT in children took place on Hilton Head Island, SC, in March 1994 and included detailed reviews of the role of BMT in the management of diseases for which it has been a subject of considerable controversy: haemoglobinopathies (thalassaemia and sickle cell disease), metabolic storage disorders and neuroblastoma. The results of BMT using marrow donors other than HLA-identical siblings were presented, including data from a number of centres on the outcome in children transplanted from mismatched family donors. Experience of the collection and transplantation of alternative sources of haemopoietic stem cells in the paediatric age group has accumulated rapidly in recent years. The results of transplantation of peripheral blood stem cells and of umbilical cord blood stem cells indicate that both approaches may soon replace BMT as first-line treatment for some malignant and non-malignant disorders in children. While gene therapy offers exciting prospects for the future, it was discussed here principally as treatment for ADA deficiency and is likely to remain a more distant, although exciting, therapeutic option for many diseases currently treated by BMT.
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PMID:Bone marrow transplantation in children: current results and controversies.Meeting, Hilton Head Island, SC, March 1994. 799 32