The CrescNet Registry Achondroplasia Module: Real-World Demographic Data and Clinical Outcomes in Untreated and Vosoritide-Treated Individuals
Authors' conclusion
The module facilitates the collection of real-world data to improve understanding of the natural history of achondroplasia and outcomes associated with interventions. Growth data from vosoritide-treated individuals were consistent with clinical trial findings and published real-world data. Longer term follow-up is ongoing.
Study context
- Population
- Individuals with genetically documented achondroplasia in the CrescNet registry achondroplasia module, 10 European countries, 32 tertiary centers
- Participants
- 186 vs 73
- Exposure
- Vosoritide-treated vs untreated
- Vosoritide-treatedn = 186
- Untreatedn = 73reference
- Outcome
- Mean height SDS (referenced to untreated European achondroplasia population), change from baseline with vosoritide
- Follow-up
- 1, 2 and 3 years after vosoritide initiation
Methodological detailsreal-world, multicentre, multinational, Data as of May 2025; pseudonymized data analysed centrally at Leipzig University Hospital by age and treatment status
- Real-world
- Multicentre
- Multinational
- Data as of May 2025; pseudonymized data analysed centrally at Leipzig University Hospital by age and treatment status
Main finding
As reported in the abstract
- Mean height SDS significantly increased from baseline at 1, 2 and 3 years in vosoritide-treated individuals
- p ≤ 0.0001
Observational data: associations, not effects.
Parts of this Brief are withheld: the automatic checks could not confirm them against the abstract.
Original publication
The CrescNet Registry Achondroplasia Module: Real-World Demographic Data and Clinical Outcomes in Untreated and Vosoritide-Treated Individuals. Horm Res Paediatr. Published 30 January 2026. doi:10.
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