Early initiation of therapy with vosoritide: case report. Description of the first Italian patient affected by achondroplasia treated with vosoritide before 2 years of age - Summary - MDSpire
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Early initiation of therapy with vosoritide: case report. Description of the first Italian patient affected by achondroplasia treated with vosoritide before 2 years of age
To present the case of the first patient in Italy treated with vosoritide for achondroplasia before the age of two.
Approach:
Case Description: A female newborn diagnosed with achondroplasia was treated with vosoritide starting at 7 months of age, following approval from the local rare diseases commission.
Diagnostic Assessment: The patient underwent various assessments including MRI and polysomnography to monitor her condition and response to treatment.
Key Findings:
The patient showed no severe adverse effects from vosoritide.
After 12 months of therapy, an improvement of +0.35 SD in length was documented according to growth curves specific for girls affected by achondroplasia.
The treatment was well tolerated and resulted in a progressive increase in length percentiles.
Interpretation:
Limitations:
The findings are based on a single case study.
Long-term effects of early vosoritide treatment remain to be evaluated.
