{
  "id": 8326689,
  "title": "Gene Therapy for Sanfilippo Syndrome Gets FDA Nod",
  "url": "https://urgent.news/2026/09/18/gene-therapy-for-sanfilippo-syndrome-gets-fda-nod",
  "topic": "health",
  "section": "Health & Medicine",
  "published": "2026-09-18T21:22:26.000Z",
  "source": {
    "name": "MedPage Today",
    "slug": "medpage-today",
    "url": "https://www.medpagetoday.com/neurology/generalneurology/123074"
  },
  "original_language": "en",
  "account": null,
  "summary": "(MedPage Today) -- The FDA on Thursday approved rebisufligene etisparvovec (Fayuvi) as the first treatment for pediatric patients with mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo syndrome type A, the agency announced Thursday...",
  "key_points": [],
  "editors_take": null,
  "illustration": null,
  "coverage": {
    "outlets": 4,
    "also_reported_by": [
      {
        "outlet": "Endpoints News",
        "title": "FDA approves Ultragenyx's gene therapy for Sanfilippo syndrome type A",
        "url": "https://urgent.news/2026/09/17/fda-approves-ultragenyxs-gene-therapy-for-sanfilippo-syndrome-type-a",
        "published": "2026-09-17T19:04:26.000Z"
      },
      {
        "outlet": "STAT News",
        "title": "STAT+: FDA approves a new gene therapy for Sanfilippo syndrome, an ultra-rare disease",
        "url": "https://urgent.news/2026/09/17/stat-fda-approves-a-new-gene-therapy-for-sanfilippo-syndrome-an-ultra",
        "published": "2026-09-17T19:55:46.000Z"
      },
      {
        "outlet": "BioPharma Dive",
        "title": "Ultragenyx prices Sanfilippo gene therapy at $4M; Bristol Myers shelves Orum drug",
        "url": "https://urgent.news/2026/09/18/ultragenyx-prices-sanfilippo-gene-therapy-at-4m-bristol-myers-shelves",
        "published": "2026-09-18T14:24:00.000Z"
      }
    ]
  },
  "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."
}