STAT+: Five years after disaster, a rare disease community gets new chance at treatment

Manage alerts for this article Email this article Share this article By Jason MastMay 11, 2026 Jason Mast[email protected]Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason on Signal at JasonMast.05. For the first two years of Joshua Jacob Gonzalez’s life, his parents, Javier and Jessica Gonzalez, suctioned saliva from the back of his mouth every five minutes. Miss one suction, his airways could clog and he could die. So, a few weeks after treatment, it was the first thing Jessica noticed. She jostled Javier, who was asleep in a hospital chair at the National Institutes of Health, awake. When was the last time he had suctioned JJ? An hour had elapsed, they realized, and their boy was breathing fine. Advertisement It was the beginning of a chapter that would prove as bittersweet for the Gonzalezes as it was miraculous. 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 FDA, gene editing, Gene therapy, rare diseases, research, STAT+ Submit a correction requestReprints Jason Mast General Assignment Reporter Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason on Signal at JasonMast.05. Recommended Exclusive May 11, 2026 STAT Plus: Medicare is spending far less than expected on new Alzheimer’s drugs Biotech May 8, 2026 The biotechs wear Prada Advertisement The Readout May 8, 2026 STAT Plus: The biotech news you need to read today The Readout May 7, 2026 STAT Plus: FDA revisits a rare cancer treatment it rejected a few months ago Biotech May 7, 2026 STAT Plus: FDA to reconsider treatment for rare cancer after its surprise rejection Subscriber Picks