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Real-world evidence of vosoritide treatment in children under 2 years of age with achondroplasia in Colombia: a cases series
By MC L, G G, GL P and 4 others.
Abstract
Abstract Introduction: Achondroplasia is the most common skeletal dysplasia and is associated with significant medical complications and impaired linear growth. Vosoritide is the first targeted therapy approved for achondroplasia; however, real-world data in children younger than 2 years of age are scarce, particularly in Latin America.
