HARTFORD, Wis. — A Hartford mother is pushing for U.S. Food and Drug Administration approval of a drug that could help her 13-year-old son who lives with a rare genetic illness.
Amy Wald emailed TMJ4 News asking to share her son Levi's story of survival. He is living with Barth Syndrome, a genetic illness caused by a random mutation that affects his breathing, heart and muscle development.
According to Amy, only 200 people live with this illness across the world.
“When you can’t do something to help your son and those other families?" she said. "That’s the tough part.”
The Walds are pleading with the FDA to approve a new drug called elamiprepitide, which was denied approval last week.
"Now that we finally have it and we're not able to have it for our boys who have Barth Syndrome, that scares the living crap out of me," Amy said. "To not be able to have it if we need it."
She fears for her son's future, noting that other mothers have lost their sons at Levi's age.
"It's very random. You don't know," she said. "It could be the simplest thing, like them getting sick with a really bad bacterial infection that goes straight to their heart. It could be anything really."
Despite his condition, Levi's parents try to provide him with as normal a life as possible. Baseball is his passion, though his condition makes physical activity challenging.
"Playing baseball with this is a little bit harder to run because you have it, you get slower," Levi said. "But it's still fun because you're with all your friends."
Amy noted that with age, Levi's condition becomes more limiting.
"As Levi gets older, it gets harder," she said. "The stamina piece gets tougher, and the energy levels go down."
In her effort to change the FDA's decision, Amy has emailed numerous legislators including Scott Fitzgerald, Ron Johnson and Tammy Baldwin.
Watch: Hartford mom fights for FDA approval of drug to help son with genetic illness
"God forbid he needs that drug too," she said. "I want him to be able to have it when and if that time is. So, I'm fighting to make sure everyone stays on it."
She hopes sharing her family's story publicly will push leaders to make a change promptly, before it's too late for Levi.
TMJ4 News requested an interview or statement from the FDA about why the drug was denied approval, to which they responded with the following statement:
"The FDA bases approval decisions on whether an application submitted to FDA for approval of the drug show that it is safe and effective for use under the conditions in its proposed labeling. This includes a benefit-risk assessment based on the totality of the data presented in each application."
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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