Sickle cell disease remains a major health problem in many African countries. The World Health Organization estimates about 515,000 babies are born with the condition each year and about 80 per cent of cases occur in Sub-Saharan Africa. In that region the disease is a leading cause of death among young children.
On 9 February the Ugandan Ministry of Health introduced a policy requiring all babies born in health facilities to be screened for sickle cell disease at birth, free of charge. Official figures put the number of Ugandan children born with the condition at about 20,000 each year, and up to 80 per cent die before the age of five because diagnosis and treatment often come too late.
At the same time regulators in high-income countries have approved new gene therapies. The US Food and Drug Administration approved Casgevy in December 2023 and the UK authorised it for use in the NHS in January. Casgevy uses CRISPR to edit blood stem cells, which are reinfused after chemotherapy, and trials showed fewer severe pain episodes. However, Casgevy costs about US$2.2 million in the United States and remains out of reach for most people in Africa.
Advocates urge investment in prevention, screening, training and local manufacturing so treatments become realistic and equitable for low- and middle-income countries.
Difficult words
- sickle cell disease — inherited blood condition causing pain and health problems
- screen — to test people for a health problemscreened, screening
- advocate — person who publicly supports a cause or policyAdvocates
- equitable — fair and equal for everyone
- stem cell — a cell that can develop into different tissuesstem cells
- chemotherapy — drug treatment that kills cancer cells
- investment — money or effort put into something for benefit
Tip: hover, focus or tap highlighted words in the article to see quick definitions while you read or listen.
Discussion questions
- How might free newborn screening change outcomes for children with sickle cell disease in Uganda? Give two reasons.
- Suggest two practical steps that could make sickle cell treatments more accessible in low- and middle-income countries.
- Do you think expensive gene therapies should become available in low-income countries? Why or why not?
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