Therapy for a rare condition in children


After 30 years of research, a therapy has been approved to treat a rare condition that leaves children susceptible to seemingly harmless infections.


Congenital athymia, where children born without a thymus gland cannot produce some immune cells in response to infection, results in an average life expectancy of around 2-3 years.


Early research at Duke University School of Medicine, USA, conducted in mice, showed that by adding small parts of the thymus tissue into the area where the thymus should be, they could start to regenerate the gland and immune cells.


Working with Enzyvant, the first human trials of the drug were a great success, with many of the initial patients now living into their 20s and recent approval by the Food and Drug Administration now means it is ready for general use in children with the condition.

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