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A new drug is showing promise as a treatment for children with a rare genetic disorder that causes short stature, according to researchers at Children's National Hospital.
The drug, vosoritide, increased growth rates by 1.8 inches per year in children with hypochondroplasia, which is caused by a mutation in the fibroblast growth factor receptor 3 gene, reports the New York Times.
"This is the first medicine that has been developed to specifically target the pathway involved in hypochondroplasia," says Dr. Andrew Dauber, chief of Endocrinology at Children's National, in a press release.
"These findings will help inform future studies of vosoritide for addressing growth disorders."
The drug, made by BioMarin, was found to be safe and well-tolerated in a clinical trial involving 24 children with hypochondroplasia who were given a daily dose of 15 micrograms of vosoritide for a year.
The children's height standard deviation score increased by 0.38 inches per year during the treatment period, and there were no serious adverse events.
hypochondroplasia is caused by a mutation in the F3 gene that makes the body produce too much of the growth factor.
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