Minnesota mother advocates on Capitol Hill for son's treatment
Minnesota mother advocates on Capitol Hill for son's treatment
WASHINGTON — A northwest Minnesota mother returned to Capitol Hill on Tuesday, March 10, to advocate for her son and other children living with Duchenne muscular dystrophy.
Cheri Gunvalson’s son, Jacob, was born with the rare genetic condition. A drug that has helped him survive, ataluren, recently fell short of Food and Drug Administration approval, halting access for patients like Jacob.
Gunvalson, of Gonvick, Minn., spoke at Capitol Hill in support of ataluren, saying the drug is critical to her son’s ability to breathe. “We are asking the FDA to provide the flexibility for our drug that it’s given to other drugs with Duchenne like Elevidys,” Gunvalson said.
Jacob has been able to graduate college, work as a social worker, and live five hours from home while managing Duchenne. His mother emphasized that losing access to ataluren would be devastating. “All these boys are going to lose this drug in the next few months, and we've already lost it,” she said. “Without it, Jacob will decline and die.”
Jacob Gunvalson, born with Duchenne muscular dystrophy, has graduated college, works as a social worker and relies on the drug ataluren to breathe.
Gunvalson has shared Jacob’s story with U.S. lawmakers for decades and is continuing the fight. She spoke alongside U.S. Sen. Ron Johnson, R-Wis., who said he plans to investigate the FDA’s reasoning for rejecting ataluren’s new drug application.
Jacob has participated in clinical trials using ataluren for more than 20 years. Although PTC Therapeutics withdrew the new drug application in mid-February after the FDA did not find sufficient evidence in trial data, Gunvalson and other parents continue to champion its safety.
“You don’t know what’s going to happen, but families deserve that choice. Our drug has been in clinical trials with hundreds of boys, and it’s proven very safe,” she said.
Cheri Gunvalson described her son’s resilience. “I once asked him, I said, ‘Jacob, why aren’t you? Why don’t you complain like your siblings?’ He says, ‘Mom, I’m not going to waste one more second on this disease than I have to.’ He’s got just a unique personality. He’s seen it all.”
The Gunvalson family no longer has access to the drug since the clinical trial ended and continues to search for options for Jacob.