{
  "id": 97226,
  "title": "After Receiving a Custom Experimental Medicine, a Teen With a Rare Genetic Disorder Walked on His Own for the First Time",
  "url": "https://urgent.news/2026/08/03/after-receiving-a-custom-experimental-medicine-a-teen-with-a-rare",
  "topic": "science",
  "section": "Science",
  "published": "2026-08-03T17:31:49.000Z",
  "source": {
    "name": "Smithsonian",
    "slug": "smithsonian",
    "url": "https://www.smithsonianmag.com/smart-news/after-receiving-a-custom-experimental-medicine-a-teen-with-a-rare-genetic-disorder-walked-on-his-own-for-the-first-time-180989230/"
  },
  "original_language": "en",
  "account": "Teenager Connor Dalby, who has a rare genetic disorder, began walking unassisted for the first time after receiving an experimental medicine tailored to his specific genetic mutation. The 17-year-old, whose mother Kelley Del Real described as a life-changing development, experienced a dramatic reduction in seizures and increased independence. His condition, affecting brain development and movement, was caused by a harmful copy of the SCN2A gene, which led researchers to create allele-selective antisense oligonucleotides (ASOs) to target the faulty DNA. Dalby's case, alongside a similar 9-year-old boy with the same genetic mutation but a different mutation, contributed to the \"n-of-one\" medicine approach. This innovative therapy, developed by the n-Lorem Foundation, demonstrates the potential for personalized medicine to reverse previously irreversible neurological issues. However, the long-term effectiveness and scalability of such treatments remain to be determined.",
  "summary": "The patient's genetic disorder causes frequent seizures and developmental delays. His progress, along with that of another boy who received a similar personalized treatment, highlights the promise—and challenges—of tailor-made drugs",
  "key_points": [],
  "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."
}