The CrescNet Registry Achondroplasia Module: Real-World Demographic Data and Clinical Outcomes in Untreated and Vosoritide-Treated Individuals
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.
Authors
- Christoph Beger (ORCID: https://orcid.org/0000-0002-1166-0368)
- Jeanne M. Pimenta (ORCID: https://orcid.org/0000-0003-3482-7770)
- Tilman Rohrer (ORCID: https://orcid.org/0000-0001-9516-7863)
- Ruth Gausche (ORCID: https://orcid.org/0000-0001-5809-6330)
- Nicole Muschol (ORCID: https://orcid.org/0000-0003-4213-7294)
- Katja Palm (ORCID: https://orcid.org/0000-0003-4383-266X)
- Katja Wechsung
- Klaus Mohnike
- Ursula Eibenstein
- Jiří Zeman
- Maria Sredkova
- Anja Reichert
- Heike Hoyer-Kuhn
- Ioana Streata
- Joachim Woelfle
- Roland Pfäffle
Publication Details
- Journal
- Hormone Research in Paediatrics
- Published
- 2026-01-30
- DOI
- https://doi.org/10.1159/000550795
- Primary Topic
- Connective tissue disorders research
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- BioMarin Pharmaceutical
- Universität Leipzig