{
  "abstract": "We know that achondroplasia is caused by variants of the FGFR3 gene and limb lengthening has been one of the only options to attain a greater height as an adult. Only recently have medical options been available such as the C-type natriuretic peptide analogues vosoritide and navepegritide. These agents bind to the natriuretic peptide receptor B to inhibit the overactive mitogen-activated protein kinase (MAPK) pathway downstream of FGFR3. They are given subcutaneously. An interesting, FGFR1–three tyrosine kinase inhibitor which down-regulates key pathways in the pathogenesis is called infigratinib and this is given in an oral form. Savarirayan R et al [NEJM 2026 DOI: 10.1056/NEJMoa2604565] have published the results of a phase 3, double blind, multicentre, placebo-controlled study in 114 children and young adults aged 3 years to 17 years, with achondroplasia. There were 75 subjects who received 0.25 mg/kg of oral infigratinib and 75 subjects who were given placebo once daily mean change from baseline to week 52 was 1.74 cm per year (95% CI (CI), 1.31 to 2.17; p<0.001) for the annualised height velocity, 0.32 (96% CI, 0.23 to 0.41; p<0.001) for the height z score, and −0.02 (96% CI, −0.06 to 0.01) for the upper-to-lower body segment ratio. Adverses effects and serious adverse effects were similar ion both groups, but no serious adverse events or adverse events lead to treatment discontinuation. So, this treatment appears to work. Lucina is sure more studies will come.",
  "authors": [
    {
      "affiliations": [],
      "name": "BMJ Publishing Group Ltd and Royal College of Paediatrics and Child Health"
    }
  ],
  "title": "Highlights from the Literature",
  "uid": "9732acbb-b9e3-56c7-89b7-10eef11725a3"
}