Promising Medication Battles the Topmost Source of Dwarfism
Key Takeaways:
- An experimental medication, vosoritide, appears to enhance growth rates in children with achondroplasia, a genetic bone disorder recognized as the primary type of dwarfism.
- A study has shown that the growth rate in height among participants surged by approximately 2.4 inches annually after taking vosoritide, similar to the average growth rates in children of standard stature.
- The primary side effects reported due to the medication were mostly mild, and it is currently being tested in a phase 3 trial with a broader group of patients.
- Achondroplasia affects approximately 1 in every 25,000 infants and is caused by an abnormality in the FGFR3 gene, often leading to complications such as spinal cord compression, spinal curvature, and bowed legs.
- The treatment offers potential not just for the treatment of health outcomes and functioning in children with the condition, but also enhanced height and accessibility to their surroundings.
An experimental medication seems to have enhanced growth rates in children diagnosed with dwarfism, as indicated by a contemporary study.
The study carried out a phase 2 trial over a span of four years and involved 35 children, ranging from 5 to 14 years, suffering from achondroplasia – a genetic bone disorder, commonly recognized as the primary type of dwarfism. The trials were conducted in multi-locations, including the United States, France, the United Kingdom, and Australia.
Role of Vosoritide
Daily doses of vosoritide, a medication that contributes to the regulation of bone growth, were administered to the children. These participants were segregated into four distinct groups, each receiving increasing doses of the treatment.
As per the study, which has been recently published in the renowned New England Journal of Medicine, the growth rate in height amongst the participants surged by approximately 2.4 inches annually. Such an increase is rather similar to the average growth rates in children of standard stature.
Significant Growth Increase
The authors of the study have confirmed a 50% increment in the growth rate among children. The study was constructed and sponsored by a pharmaceutical organisation, BioMarin Pharmaceutical, which was also responsible for data analysis.
Side Effects and Further Exploration
Reported side effects due to the medication were mostly mild, the researchers have assured. Currently, vosoritide is under testing within a phase 3 trial involving a broader group of patients between 5-18 years of age.
Understanding Achondroplasia
Ravi Savarirayan, the primary author of the study and a clinical geneticist at Murdoch Children’s Research Institute in Melbourne, Australia, elaborates on the cause of achondroplasia. He compares it to a plant being watered excessively that hampers growth. He asserts that the medication essentially “kinks the hose” enabling the correct water supply and resuming regular growth.
Achondroplasia affects approximately 1 out of every 25,000 infants. It is instigated by abnormality in the FGFR3 gene that restricts the growth of bones in the limbs, spine, and the base of the skull. Children diagnosed with the condition often face common complications such as spinal cord compression, spinal curvature, and bowed legs, with about half of them requiring spinal or other forms of surgery.
In the words of Savarirayan, “This study is the inaugural report of a possibly game-changing, precise therapy for children with achondroplasia. We envisage not just an improvement in their health outcomes and functioning, but also an enhancement of their height and accessibility to their surroundings.”
Additional Information
For more insights on achondroplasia, visit the U.S. National Library of Medicine.
