In a medical first, drugs have reversed an inherited disorder in the womb
The injection of a protein at just the right moment during pregnancy appears to have spared a set of twins—and one other child—from being born without sweat glands.
The daring pregnancy intervention is being described as the first time a drug has been used to treat a developmental disorder in utero.
The experiment, described in a case report today in the New England Journal of Medicine, took place in Germany in 2016 at a clinic that specializes in rare, inherited skin diseases—particular one called XLHED, in which patients are born with fang-like front teeth and without the ability to sweat.
The problem: their bodies don’t produce a specific protein required to make sweat glands.
The German clinic, at the University of Erlangen-Nürnberg, had already participated in a clinical study testing a protein replacement treatment in young children.
But the drug did nothing for the children, the study was abandoned, and the drug maker, Edimer Pharmaceuticals, shut down.
