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US Approves Drug To Improve Growth In Children With Dwarfism

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The US Food and Drug Administration on Friday approved for the first time a drug to improve growth in children with the most common type of dwarfism.

 

Voxzogo, manufactured by American pharmaceutical company BioMarin, is authorized in the US to treat kids older than five with achondroplasia whose growth plates — zones of cartilage at the end of long bones — are still open, meaning they still have the potential to grow.

 

“With this action, children with short stature due to achondroplasia have a treatment option that targets the underlying cause of their short stature,” said FDA endocrinologist Theresa Kehoe in a press release.
Achondroplasia is a genetic bone growth disorder that blocks the changing of cartilage to bone. Adults with the condition grow to be about four feet tall (1.2 meters) on average.

It can cause health issues including breathing problems, exaggerated spine curvature, obesity, and recurrent ear infections, according to the National Institutes of Health. Life span is usually near normal.

“As a parent of a child with achondroplasia, I see the availability of treatments that impact bone growth as an important step forward,” Amer Haider, the co-founder of nonprofit group Growing Stronger, said in the BioMarin statement.
Growing Stronger is dedicated to improving the quality of medical care for little people through supporting research.

 

But growing taller is not necessarily the goal of all with achondroplasia.
When BioMarin shared preliminary test results in 2015, dwarfism advocacy organization Little People of America (LPA) emphasized that it aims to celebrate dwarfism as a “valuable contribution to the diversity of the human condition.”

“While we encourage individuals and families to make the decision that is best for them, we stress that emerging treatments are not necessary for people with dwarfism to live engaging, healthy, productive lives,” the LPA statement said.

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