6-Year-Old Gets Life-Changing Drug Weeks After FDA OK
Rory Jaskulski received a newly approved treatment for rare Hunter syndrome just three weeks after FDA approval, transforming his family's outlook from planning a funeral to imagining his future. The New Berlin boy is among the first patients to access a medication the community waited nearly 20 years to see approved.
Six-year-old Rory Jaskulski just became one of the first children in America to receive a groundbreaking treatment for Hunter syndrome, a rare genetic disease that was stealing his future.
Just three and a half weeks after the FDA approved the new medication from manufacturer Denali, Rory sat for his first infusion on April 17. His mother, Kylie Jaskulski, had reached out to media last month to advocate for the drug's approval, never imagining it would happen so quickly.
Hunter syndrome prevents the body from breaking down certain molecules, causing them to build up and create harmful deposits. The progressive disease leads to physical disabilities and cognitive delays, with a life expectancy of only 10 to 20 years. Rory also has autism and is non-verbal.
The speed of Rory's access to treatment surprised even his family. Kylie expected months of waiting after approval, time her son didn't have to spare.
"We needed it to happen before Rory got worse, so the fact that this got approved before Rory progressed, it's nothing short of a miracle," she said.
The approval marks the first new FDA-approved treatment option for Hunter syndrome in nearly two decades. Denali representatives told TMJ4 that stories like Rory's remind them why advancing treatments for neurodegenerative and lysosomal storage disorders matters so deeply.
Why This Inspires
Kylie's transformation from despair to hope captures what medical breakthroughs mean beyond the science. She went from planning her young son's funeral to wondering what his future might hold.
Now she's thinking about possibilities she never dared imagine. The family still has a long road ahead as they monitor how well the medication works, but the shift in perspective changes everything.
Kylie's thoughts have turned outward too. As other gene therapies move through the FDA approval process, she's praying for families still waiting for their own miracles.
"Every one of those children deserve to have the same opportunities that my son has now been given."
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Based on reporting by Google: new treatment approved
This story was written by BrightWire based on verified news reports.
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