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The β-haemoglobinopathies are the most prevalent inherited disorders worldwide. Gene therapy of β-thalassaemia is particularly challenging given the requirement for massive haemoglobin production in a lineage-specific manner and the lack of selective advantage for corrected haematopoietic stem cells. Compound β(E)/β(0)-thalassaemia is the most common form… (More)
Considering the high financial risks involved in introducing new services, and in meeting certificate-of-need requirements, health delivery systems should use a more systematic approach involving long-range planning.