Publication details
Oral Infigratinib Therapy in Children with Achondroplasia
| Authors | |
|---|---|
| Year of publication | 2025 |
| Type | Peer-reviewed scientific article |
| Magazine / Source | New England journal of medicine |
| MU Faculty or unit | |
| Citation | |
| web | https://www.nejm.org/doi/full/10.1056/NEJMc2504219 |
| Doi | https://doi.org/10.1056/NEJMc2504219 |
| Keywords | achondroplasia; infigratinib; pediatric therapy; FGFR inhibition; clinical treatment |
| Description | Savarirayan et al. report that oral administration of the FGFR inhibitor infigratinib to children with achondroplasia improves longitudinal growth and skeletal proportions. Their study represents an important advance over current vosoritide therapy in terms of efficacy (higher annualized height velocity) and administration (oral vs. daily injections). However, the boundaries of achondroplasia therapy can be pushed even further. |