# FDA approves a new gene therapy for Sanfilippo syndrome, an ultra-rare disease

> **Newsylist Open Intelligence Dossier** · First detected: 2026-09-18 19:29 UTC · Category: Business · Sources: 5 · Current Velocity: 3.35

## Key People, Organizations & Locations
FDA, Sanfilippo

## Multi-Source Evidence Table
| Source Outlet | Headline | Published (UTC) | Verification URL |
|---|---|---|---|
| Yahoo Finance | RARE Stock Up 13% on FDA Approval of Sanfilippo Syndrome Gene Therapy | 2026-09-17 19:59 | [Source Link](https://news.google.com/rss/articles/CBMikAFBVV95cUxNUUdkel82TlBSZ1JuMjg3eDY1Z0w4anVOcW1xaHBlMWVmSnFMVGhrekUyWmZaMVNOYWc4ckdXT0VVV1FuRzhoeTZvVm5KOW5wMjU2YkZOb1BwWjY4R21mRWw0MHpDVmV1WnBjT0RFeFJlSEdYVGZ1c2xyNGVGa0tFb2diS3puVm1yMHdYVnZTWFA?oc=5) |
| Endpoints News | Updated: Ultragenyx’s new gene therapy will cost $3.95 million | 2026-09-17 19:59 | [Source Link](https://news.google.com/rss/articles/CBMilwFBVV95cUxQR1NVX2lUb0xCYWlFckhWUFdVUnhrVXFDVkVWaHRsZXM5LW9kTG9fTGJVQk03R0p6TmJvR3J2QzBPREFSNHdhdHgwX0FzT1JzLWZHZEctOEtZb2hqY3NQXzRSd1RXVXFVTG9JbWl1SUJOZ2xTeFZ0WWx2X0czQjN0ZE41U0tzcnBmODZMRGszTWR1U3lXQ3VF?oc=5) |
| Reuters | Ultragenyx sets $3.95 million price for rare disease gene therapy | 2026-09-17 19:59 | [Source Link](https://news.google.com/rss/articles/CBMixAFBVV95cUxOeE90bDVTdWlBazlIN1NQLXJHbklsNk1MQ09JTy1tTHdDd0Jja0tOY09ZeEJJQjJnVW52TzVFa2NSSmdsMHhOQXJiV2VPR0RPYktma2k2OHUtTHItY2F2OEs0M0ZIeTJsQmltNWFpUU8tZDlvMUFtY1F4U0ZfaXFIb3kwaEh3ei1qdmtoLXVMMDZJX2lLemIzblhHWWJ0Nkd2RFYwbm5qempENjFPYk5GYkZhUlNnMFNLZGJYTXFfcTR4UmpC?oc=5) |
| Yahoo Finance | Ultragenyx Gets A ‘Much-Needed Win,’ Says Cantor – Sees $400M Opportunity After FDA Approves Neurodegenerative Disease Therapy | 2026-09-17 19:59 | [Source Link](https://news.google.com/rss/articles/CBMilwFBVV95cUxOWjJxaWN3Z2xERmlwcURhQW82RmFFQUFEWXpNSFlNVk5wSWg5X1ozeUV3aFNISHkxLXkza1VHS3pobVpkYlJMalJVMXg5WV9XaHMzeUxidzE1cS1HRmNzVXpaU21yQ2dLVTJEbnhjQmllUUVzQXBkYVlyM2p3cS1TVC1PdmppLUxuUUtrVTNKM2JObUhPaEZr?oc=5) |
| Fierce Pharma | FDA approves Ultragenyx’s gene therapy Fayuvi for rare neurodegenerative disorder | 2026-09-17 19:59 | [Source Link](https://news.google.com/rss/articles/CBMisAFBVV95cUxOaF9GYmFoVU84czdXV2FhSjdNS0VhNHRCTklYeEFqNjlpWU0zYXFjMXpxNEFkeXVLREhiVmFvenJyY0ZLZTNRRnZQc2Y3Tjl2ODZlalpxUjFhZkl5aGZIX2F5QVJYaFZyT3VfaW9FSEZBTi1aNjZUWE1yeDBCazBERVRSajVOeFdUQXVVMDhuajlYZWUwRkVPak5iUGRGcGlTLVhTNzVSVmdRaUJZeERlNg?oc=5) |
| STAT | FDA approves a new gene therapy for Sanfilippo syndrome, an ultra-rare disease | 2026-09-17 19:59 | [Source Link](https://news.google.com/rss/articles/CBMijwFBVV95cUxQdVl6aVRmQVhsZWRGNDNKUmVuOXo0Q1dkN3dDYkF5bWZNd29RblRBUnl3OXEtSlowdGx0d2dRZERsYm9LZUVZRFA5eC1pUnRUaWZoVHpQMndDdmt1eC1RQlU5WTFtRlp3SnJPTlZUM3VXcFZ4enZEbzJ5dEhJd0drdVROUUpodHZoUDY5akZ6SQ?oc=5) |

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