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The problem
Intervention: Treatment with vosoritide (C-type natriuretic peptide analog), either as initial therapy or following transition from growth hormone therapy
Who it was tested with: 22 children with achondroplasia at a single Japanese institution; Group A (n=13, median age 2.10 years) receiving vosoritide alone; Group B (n=9, median age 10.1 years) transitioning from prior growth hormone treatment.
Experience from people who deliver programmes: stories, case studies and lessons learned. Valuable for understanding how and why, and for spotting what to test next, but not a measure of effect.
"We conducted a retrospective study of 22 children with ACH treated with vosoritide for over 6 months at a single institution...Patients were divided into two groups: GH-naïve group (Group A, n = 13), receiving vosoritide alone, and a switch group (Group B, n = 9), who transitioned from prior GH treatment." The study measures growth at multiple timepoints pre- and post-intervention but lacks a concurrent comparison group, making it a pre-post design that demonstrates treatment effects but not superiority over alternatives.
Small sample size (n=22), single-center retrospective design, no concurrent control group, varying follow-up durations, and lack of randomization limit causal inference. Results may not generalize to other populations.
Across countries and designs
1 study · 1 country
