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1
Achondroplasia is the most common skeletal dysplasia, characterized by disproportionate short stature and reduced linear growth, especially in infancy.
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2
Vosoritide is the first approved drug to improve linear growth in children with achondroplasia, now approved for use in infants from 4 months of age.
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3
In Italy, vosoritide was initially reimbursed only for children over 2 years old until the end of October 2025, when it was extended to younger patients.
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The first patient in Italy treated with vosoritide under 2 years of age began therapy at 7 months, showing no severe adverse effects.
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5
After 12 months of vosoritide therapy, the patient demonstrated an improvement of +0.35 SD in length, consistent with previous studies.
