{
  "id": 5216668,
  "title": "Ultragenyx’s Angelman therapy fails Phase 3 trial in setback for",
  "url": "https://urgent.news/2026/09/03/ultragenyxs-angelman-therapy-fails-phase-3-trial-in-setback-for",
  "topic": "health",
  "section": "Health & Medicine",
  "published": "2026-09-03T01:35:17.000Z",
  "source": {
    "name": "Endpoints News",
    "slug": "endpoints-news",
    "url": "https://endpoints.news/ultragenyxs-angelman-therapy-fails-phase-3-trial-in-setback-for/"
  },
  "original_language": "en",
  "account": null,
  "summary": "Ultragenyx's experimental therapy for Angelman syndrome, a rare neurodevelopmental disease, has failed a Phase 3 trial. The therapy, GTX-102, an antisense oligonucleotide, showed no benefit compared to a sham treatment.\n\nThe failure is a significant setback for the search for a treatment for Angelman syndrome, which causes severe intellectual disabilities and developmental delays. According to STAT News, the therapy had shown powerful results in early trials, raising hopes for families affected by the condition.\n\nThe news is also a blow to Ultragenyx's business, as the company had been relying on the Angelman drug as a path to profitability, despite having multiple approved medicines for ultra-rare diseases.",
  "key_points": [],
  "editors_take": null,
  "illustration": null,
  "coverage": {
    "outlets": 2,
    "also_reported_by": [
      {
        "outlet": "STAT News",
        "title": "STAT+: Ultragenyx drug to treat Angelman syndrome, a rare disease, fails late-stage trial",
        "url": "https://urgent.news/2026/09/02/stat-ultragenyx-drug-to-treat-angelman-syndrome-a-rare-disease-fails",
        "published": "2026-09-02T20:37:24.000Z"
      }
    ]
  },
  "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."
}