Suffering from achondroplasia, a 9-year-old girl was able to benefit from treatment against dwarfism, as part of a clinical trial conducted by Necker Hospital and the Imagine Institute of Genetic Diseases.
They are only twelve children in the world to have had access to this new treatment. Since her birth, 9-year-old Amandine has suffered from achondroplasia, the most common form of dwarfism. She is currently 1.03 meters tall, 30 centimeters shorter than most children her age. Living in Rouen, she became the first child in France to test this molecule, infigratinib, intended to make her grow, as reported by Le Parisien.
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For ten days and for at least two years, this child will swallow seven pellets daily. While the average height of women with achondroplasia is 1.24 m in adulthood, researchers who follow her hope for a gain of 10 to 20 centimeters during these two years.
A glimmer of hope
The consequences of this constitutional bone disease, which affects 2,400 people in France, are very restrictive, underlines Le Parisien. This is characterized by an abnormality that affects the growth of cartilages, especially those of the limbs and also creates a risk of compression of the skull, spine and back problems, which earned Amandine a daily life with a corset until she was three years old.
This young boy with dwarfism has a message
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If his parents, Valérie and Pascal, consider this treatment as a “chance”, they nevertheless remain aware that infigratinib is not miraculous: “It will not cancel the disease, but it will help it: every centimeter gained is a victory”, they told the daily.