ArticleNews reportPHN-05002026-11-01 06:39 CT
FDA Approves Gene Therapy After Family Funds Research
Source · Cirebon Times
TLDR
Cirebon Times reports that the FDA approved Ultragenyx’s gene therapy Fayuvi for Sanfilippo syndrome after patient families helped fund its development. The O’Neill family’s campaign raised $2 million to support the first human trial; the article says the therapy is priced at nearly $4 million.
01—Key points
- 01The article reports that Ultragenyx acquired the therapy’s rights in 2022 and secured FDA approval in September.
- 02Eight patient foundations helped sustain research leading to the first human trial in May 2016.
- 03Eliza O’Neill was the first trial participant; the article says her lost speech has not returned.
Why it matters Editorial
The report highlights patient-funded development of a rare-disease gene therapy, rather than a peptide-based treatment.
Summary generated with AI and reviewed by an editor. The original source governs; summaries can contain errors. Information only — not medical advice. Full disclaimer.
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