{
  "id": 6281706,
  "title": "These life-saving treatments were considered ‘too small’ to fund. Until now.",
  "url": "https://urgent.news/2026/09/08/these-life-saving-treatments-were-considered-too-small-to-fund-until",
  "topic": "science",
  "section": "Science",
  "published": "2026-09-08T13:00:00.000Z",
  "source": {
    "name": "Big Think",
    "slug": "big-think",
    "url": "https://bigthink.com/sponsored/these-life-saving-treatments-were-considered-too-small-to-fund/"
  },
  "original_language": "en",
  "account": "When 10-month-old Noa Greenwood was diagnosed with Canavan, her parents Lee and Lori faced an unimaginable future. Canavan is a rare, inherited neurodegenerative disease caused by mutations in the ASPA gene, and most children with it fail to develop beyond the abilities of a six-month-old. The moment Noa received her diagnosis, a trial for Canavan therapy emerged on clinicaltrials.gov, and she became one of the first children to receive the treatment. Within months, Noa began achieving milestones her parents were told she'd never attain. Just a few years ago, this scenario would have been unthinkable. Rare diseases often presented too small a market for traditional pharmaceutical companies to fund research, even when potential cures had been discovered by medical researchers. To address this issue, BridgeBio devised a new model based on portfolio theory: instead of investing in a single rare disease treatment at a time, they spread the risk across 150 different treatments. Noa's story offers hope not only to those affected by Canavan disease but also to the 450 million people worldwide who suffer from rare diseases. This development is highlighted in the video \"These life-saving treatments were considered 'too small' to fund. Until now.\" on Big Think.",
  "summary": "When 10-month-old Noa Greenwood was diagnosed with Canavan, her parents Lee and Lori were confronted with an idea of a future they’d never prepared for. Canavan is a rare, inherited neurodegenerative disease caused by mutations in the ASPA gene. Most children with it never develop beyond the abilities of a six-month-old. The very day that Noa was diagnosed, a trial for Canavan therapy was posted…",
  "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."
}