New study links oral therapy to growth in children with dwarfism

AURORA, Colo. (KRDO) -- A new study shows children with achondroplasia, the most common form of dwarfism, grew more than two centimeters per year on a new oral therapy, according to health officials.

Children's Hospital Colorado was one of 27 sites across ten countries to participate in the study, which compared children on the oral medication to those on a placebo.

Achondroplasia occurs in one out of every 26,000 to 40,000 and is caused by genetic changes that affect bone growth, differences in the shape of the spine and limbs and proportionality, according to the hospital.

The trial was conducted at Children's Hospital Colorado and the University of Colorado Anschutz School of Medicine. The new therapy, The trial was conducted at Children's Hospital Colorado and the University of Colorado Anschutz School of Medicine. The new therapy, infigratinib, is sponsored by BridgeBio Pharma.

According to the hospital, 114 children ages three to 18 with achondroplasia were treated either with oral therapy or a placebo over one year. The results showed those given the oral therapy grew 2.10 centimeters per year compared to those on the placebo.

The hospital says researchers see the results as promising, but need more time to understand long-term effects to overall health, physical function and quality of life. No serious side-effects were reported during the study.

The participants are continuing on to a longer-term study.

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