{
  "id": 5541134,
  "title": "New treatment approach for children with short-limbed dwarfism",
  "url": "https://urgent.news/2026/09/04/new-treatment-approach-for-children-with-short-limbed-dwarfism",
  "topic": "science",
  "section": "Science",
  "published": "2026-09-04T11:20:03.000Z",
  "source": {
    "name": "Medical Xpress",
    "slug": "medical-xpress",
    "url": "https://medicalxpress.com/news/2026-09-treatment-approach-children-short-limbed.html"
  },
  "original_language": "en",
  "account": "A phase 3 trial conducted by UCLA Health has shown promising results for a new treatment approach in children with achondroplasia, a genetic condition causing disproportionate short stature. The study, published in the New England Journal of Medicine, involved 114 children aged 3-17 who were randomly assigned to receive either once-daily oral infigratinib or a placebo for 52 weeks. The results indicated that the participants receiving infigratinib experienced a significantly greater increase in growth from baseline compared to those on placebo. Dr. Isidro B. Salusky, a distinguished professor of pediatrics at UCLA, highlighted the importance of early intervention, as the drug's effects are most prominent when treatment begins at an early age. The oral administration of infigratinib offers convenience, particularly for infants who are likely to benefit the most from this pharmacologic intervention. Salusky acknowledged the study's contribution to addressing a significant unmet need for patients with achondroplasia, emphasizing the value of international collaborations in studying therapeutic interventions for rare genetic disorders affecting bone.",
  "summary": "A phase 3 trial is showing \"a potentially giant step forward\" in treating children with achondroplasia, UCLA Health's Dr. Isidro B. Salusky wrote in an editorial published in the New England Journal of Medicine (NEJM).",
  "key_points": [
    "114 children aged 3-17 participated in the study, receiving either infigratinib or placebo.",
    "Infigratinib treatment led to significantly greater growth increase compared to placebo."
  ],
  "editors_take": null,
  "illustration": null,
  "coverage": {
    "outlets": 1,
    "also_reported_by": []
  },
  "ai_generated": true,
  "disclaimer": "Summaries, key points and the editor’s take are written by software from other outlets’ reporting and may contain errors — always check the linked original."
}