A New Berlin mother is waiting for the FDA to approve a new drug that could save her son from a fatal genetic disorder.
Rory Jaskulski is a kindergarten student at Orchard Lane School. He is known for his bright smile and energy.
Orchard Lane School Principal Royce Linder sees Rory Jaskulski's joy every day.
Watch: New Berlin mother pleads for FDA approval of a new drug to save her son from terminal Hunter Syndrome
"Really, when it comes down to it, he’s just a kindergarten student. He wants the same thing out of kindergarten that every kindergartener wants. He wants friends, he wants community, he wants belonging," Linder said.
When he was 4 years old, Rory Jaskulski was diagnosed with Hunter Syndrome. The genetic disorder impacts the ability to produce an enzyme that breaks down molecules. When the molecules are not broken down, they become harmful and are deposited back into the body.
The disease leads to physical disabilities and cognitive delays, like the autism that has left Rory Jaskulski non-verbal.
"It was heartbreaking, I got that diagnosis, and I wasn’t anticipating it," Kylie Jaskulski said.
Over the past two years, Kylie Jaskulski has learned a lot about the disease.
"His life expectancy is between 10-20 years as of right now with no better treatments available on the market," Kylie Jaskulski said.
Because of a treatment on the horizon, Kylie Jaskulski is full of hope. On April 5, drug company Denali is hoping for FDA approval on a new drug that would go deeper than Rory Jaskulski's current treatment. The drug crosses the blood-brain barrier into the central nervous system, hopefully treating the progressive aspects of the disease.
"I think it’s just really important for the FDA to do their job and to look at the situation that these kids are in; they don’t have any other option, there’s nothing else. It’s either this or they’re going to die from this disease," Kylie Jaskulski said.
Recently, the FDA rejected other treatments that used gene therapy to treat Hunter Syndrome. While Denali's drug is not a gene therapy, approval is not a certainty, and a rejection could mean years of delays.
"The truth of the matter is, if this drug does not get approved, in April, it won’t come in time to save Rory, and that’s really hard," Kylie Jaskulski said.
For now, Kylie Jaskulski can only wait and hold her son tight.
This story was reported on-air by a journalist and has been converted to this platform with the assistance of AI. Our editorial team verifies all reporting on all platforms for fairness and accuracy.
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