November 14th 2024.
New research led by an Australian team has revealed that children with dwarfism may have a chance to increase their height and improve their overall health with a simple daily tablet. Dwarfism, also known as achondroplasia, is a genetic condition that affects about one in every 20,000 babies born in Australia. It is characterized by short arms and legs, a larger head, and a regular-sized torso, caused by a genetic mutation.
Although many individuals with achondroplasia can live long lives, they are at risk for various health complications such as spinal cord compression, sleep apnea, respiratory blockages, and ear infections. In fact, children with achondroplasia are 50 times more likely to pass away before the age of five compared to their peers.
The new international study, led by the Murdoch Children's Research Institute, involved 72 children aged three to 11 from various countries including Australia, the UK, the US, Spain, France, and Canada. These children were given a daily dose of the drug infigratinib, which was found to increase their growth by an average of 2.5 centimeters per year over an 18-month period. This not only led to an increase in overall height, but also improved the upper-to-lower-body segment ratio.
Although some minor side effects were observed, none of the children had serious reactions to the medication. According to Professor Ravi Savarirayan, who led the study, "Our new research has shown that infigratinib is a safe and effective drug for increasing growth in children with achondroplasia. This could potentially fill the need for an oral medication for those with this condition, especially for those who cannot tolerate daily injections."
Currently, the only approved treatment for achondroplasia in Australia is vosoritide, which was added to the Pharmaceutical Benefits Scheme last year. While it helps with bone development and growth, it requires a daily injection. The new pill, infigratinib, has shown promising results in improving growth and may present a more convenient option for those with achondroplasia.
Further research is now underway to assess the safety and effectiveness of the new medication, including its potential benefits for children under the age of three. The full results of the study have been published in the New England Journal of Medicine, providing hope for children with achondroplasia and their families.
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