Lorna Knight: The Next Frontier in Sickle Cell Disease Is Access
Lorna Knight, Associate Doctor at Canada’s Medical Clinic, shared a post on LinkedIn:
“The cure exists. The access does not.
One of the most remarkable stories in medicine today is unfolding in sickle cell disease.
For decades, treatment focused on managing symptoms, reducing pain crises, and preventing complications.
Today, gene-editing therapies such as Casgevy have shown that a functional cure is possible for some patients.
That is extraordinary science.
But it also raises a difficult question.
Approximately 75% of people born with Sickle Cell Disease are born in sub-Saharan Africa.
Yet the patients most likely to access these advanced therapies are in high-income countries.
Reality:
Curative therapies now exist, but many patients still lack access to basic interventions such as newborn screening, hydroxyurea, specialist care, and reliable blood transfusion services.
Meaning:
The bottleneck in healthcare innovation is no longer always scientific discovery. Increasingly, it is access, infrastructure, affordability, and implementation.
Strategy:
While gene therapies represent the future, expanding newborn screening, improving access to hydroxyurea, strengthening healthcare systems, and using AI to support diagnosis may have the greatest impact today.
The scientific breakthrough has happened.
The next challenge is ensuring that where a child is born does not determine whether they can benefit from it.
What do you think will have the greatest impact on sickle cell outcomes over the next decade: gene therapy, healthcare infrastructure, or AI-enabled diagnosis and care pathways?”
Stay updated with Hemostasis Today.
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