A Phase 3 clinical trial has demonstrated that the drug vosoritide significantly increases linear growth in children with hypochondroplasia, a rare genetic skeletal condition causing short stature. The study, published in NEJM Evidence, revealed that after 52 weeks of treatment, children receiving once-daily injections of vosoritide achieved an annualized growth velocity difference of 2.33 cm/year compared to those on a placebo. Led by Dr. Andrew Dauber from Children's National Hospital, this multi-country trial provides promising evidence for the first potential targeted therapy for a condition that previously lacked direct disease-modifying treatment options.
Link to article: https://evidence.nejm.org/doi/pdf/10.1056/EVIDoa2600257
Children with hypochondroplasia experience significantly higher rates of neurodevelopmental and brain structural abnormalities than previously recognized. A recent cohort study reveals high prevalences of hippocampal malrotation and elevated needs for special educational support, challenging the view that the condition impacts only skeletal development. Read the full study at Developmental Medicine & Child Neurology
A commentary to the study was published by Janet Legare, MD highlighting that 71% of affected children require special educational support, prompting recommendations for comprehensive monitoring of neurodevelopmental and cognitive outcomes.
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