Eine vielversprechende tägliche Tablette steigert wirksam die Körpergröße und verbessert das proportionale Wachstum der Gliedmaßen bei Kindern mit Achondroplasie, der häufigsten Form von Zwergwuchs | Die Ergebnisse könnten Kindern die Notwendigkeit einer täglichen Injektion zur Wachstumsförderung ersparen.

From the article: [The study](https://doi.org/10.1056/NEJMoa2411790), sponsored by BridgeBioPharma Inc, involved 72 children with achondroplasia from Australia, the UK, the US, Spain, France and Canada. It reported the medication was effective at increasing the rate of growth by 2.5cm a year for up to 18 months during treatment, increasing overall height and improving the upper-to-lower-body segment ratio. Participants experienced minor side effects from the treatment but none had serious adverse reactions.

Currently, the only approved therapy for children with achondroplasia in Australia is vosoritide, a daily injection, which was added to the PBS last year. MCRI is the largest vosoritide clinical trial site in the world. Professor Savarirayan and his research team have previously shown how the drug improves bone growth development in patients as young as four months up until 18 years of age.

“Our new study found that infigratinib is a safe and effective drug that increases growth in children with achondroplasia and could meet a need for an oral medication for those with this condition,” Professor Savarirayan said. This is especially important for those children who can’t tolerate daily injections and in parts of the world where oral medications are more practicable than injections.”