Efficacy and safety of Vosoritide in achondroplasia: A systematic review and meta-analysis
Growth hormone-mimicking therapy safely improves height in children with achondroplasia over standard care alone.
This systematic review confirms vosoritide's efficacy in improving growth parameters in achondroplasia with a favorable safety profile. Longer-duration studies are needed to establish effects on medical complications.
What the study was
- Study design
- Systematic review and meta-analysis
- Population
- Achondroplasia patients aged 3 months to 18 years (N=156)
- Sample size
- 156
- Category
- Treatment Innovation
- Maturity
- Validated
- Journal
- Indian Journal of Medical Research
Why it surfaced
SR/MA of established rare disease therapy. High design quality but low novelty (vosoritide already approved).
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