STAT+: The quest to save Grace — and clear the way for rare disease patients everywhere

Manage alerts for this article Email this article Share this article By Jason Mast and Matthew HerperJuly 9, 2026 Mast and Herper have covered rare disease issues for a combined three decades. As part of the reporting, Mast spent two days in the Bay Area with Matt Wilsey and Grace. Jason Mast[email protected]Jason is a general assignment reporter, with particular focus on genetic medicine and rare disease. Confidential tips can be sent on Signal at JasonMast.77.Matthew Herper[email protected]Matthew Herper covers medical innovation — both its promise and its perils. Highlights Matt Wilsey has spent a decade and $70 million trying to develop a cure for his daughter Grace, who faces a fatal, ultra-rare disease. His company has now treated 10 patients, including Grace, in a clinical trial but is out of money and still doesn’t have what the FDA says it needs for approval. He is pressing the agency anyway, setting up a pivotal test case for regulators and for anyone developing treatments for patients facing deadly rare diseases. SAN FRANCISCO — Matt Wilsey adjusted the plastic tube coming out of his 15-year-old daughter’s stomach and tried, again, not to think beyond the next 15 minutes. His job was to be there with Grace and let his wife, Kristen, rest. He could not think about the future. He could not wonder, again, if he had made the right choice or if his daughter would survive. Three weeks before, Grace received a gene therapy meant to save her life and spare her further harm from NGLY1 deficiency, an ultra-rare genetic condition that came with a cascade of profound developmental challenges, preventing her from ever speaking or walking with ease. Advertisement Wilsey fashioned the therapy himself. He did not sit in the lab. He did not inject the rats or slice open their brains. But he had hired the scientists who did. He recruited advisers, including Nobel Prize winners, brought together the families of other children diagnosed with the condition, and pulled together an A-team of investors and donors. Through it all, he was sustained by his devout Catholic faith. He came to accept that Grace would never live an independent life. But he hoped the drug would allow her to live longer, maybe even say a few words. And he believed the game plan he wrote might serve as a guide to curing hundreds of other rare diseases. Advertisement “We carry the hopes of many,” he wrote to his staff once. “I’m not just talking about NGLY1 families. I receive emails, calls, and texts from professionals and other advocates. They are blown away by what we have accomplished and hope we are an ice breaker for them. Our trial has the potential to really boost / save a decimated field.” Then the drug meant to save Grace’s life landed her back in the hospital, feebler than she had ever been. He sat beside his daughter, with her soft eyes and long braided hair, her face all puffed up, and prayed her condition would improve. For a father and his sick child, it was a matter of life and death. But the entire pharmaceutical industry was watching, too. STAT+ Exclusive Story Already have an account? Log in This article is exclusive to STAT+ subscribers Unlock this article — plus in-depth analysis, newsletters, premium events, and news alerts. Already have an account? Log in Monthly $39 Totals $468 per year $39/month Get Started Totals $468 per year Starter $30 for 3 months, then $399/year $30 for 3 months Get Started Then $399/year Annual $399 Save 15% $399/year Get Started Save 15% 11+ Users Custom Savings start at 25%! Request A Quote Request A Quote Savings start at 25%! 2-10 Users $300 Annually per user $300/year Get Started $300 Annually per user View All Plans To read the rest of this story subscribe to STAT+. Subscribe Log In biotechnology, drug development, FDA, Gene therapy, Pharmaceuticals, rare diseases, STAT Submit a correction requestReprints Jason Mast General Assignment Reporter Jason is a general assignment reporter, with particular focus on genetic medicine and rare disease. Confidential tips can be sent on Signal at JasonMast.77. Matthew Herper Senior Writer, Medicine, Editorial Director of Events Matthew Herper covers medical innovation — both its promise and its perils. Recommended Politics July 8, 2026 STAT Plus: RFK Jr. plans to create list of injuries caused by Covid-19 vaccines Health July 8, 2026 Bryan Johnson’s chronic disease is notoriously difficult to diagnose Advertisement The Readout July 8, 2026 STAT Plus: Europe will accelerate its review of RevMed’s drug Biotech July 8, 2026 STAT Plus: Prime Medicine claims victory in dispute over gene-editing technology with Beam Therapeutics Biotech July 7, 2026 STAT Plus: FDA approves Vera Therapeutics kidney disease treatment Subscriber Picks