Family says insurance won’t approve life-altering Duchenne muscular dystrophy treatment for child

WOODBURY, Minn. (FOX 9) - Even the simplest of exercises can cause muscles to break down for a 4-year-old boy from Woodbury with a rare and serious genetic condition.

Liam Schroeder’s family and doctor say a new treatment could potentially keep him from spending the rest of his life in a wheelchair, but his parents can’t get their insurance company to approve it.

"He was having some issues climbing stairs. He was having issues just getting up," said his mom, Joan Schroeder.

Duchenne muscular dystrophy diagnosis

Two years ago, a blood test showed he was having rapid muscle breakdown. That led Dr. Jamie Eskuri, a neurologist with Gillette Children’s, to make a difficult diagnosis: Duchenne muscular dystrophy, which is a rare genetic disorder mainly affecting boys that causes muscle loss.

"I think it was because of the astuteness of his parents … realizing that there were some subtle issues going on. And he was very blessed to be working with an amazing physical therapist who identified some early red flags," Eskuri said.

She told Liam’s parents he would progress as a normal child until age 6 or 7, then start to decline.

"(We were told) he would be in a wheelchair by his early teens and hopefully make it to his 30th birthday," Joan Schroeder said.

Joan and Jon Schroeder made peace with that as much as they could, moving into a new one-story home in Woodbury and planning for the future. 

"We moved into this house, which could grow with him as his mobility declines. And we were just set for, I guess, a grim future," Jon Schroeder said.

One-time, $3.2 million gene therapy treatment 

Then, hope came in June when Elevidys, the first and only gene therapy approved for Duchenne was given the green light by the U.S. Food and Drug Administration for 4- and 5-year-olds. Liam turned 4 in July.

"Things were lining up so perfectly that we thought, ‘OK, this is just meant to be,’" Jon Schroeder said.

But since then, they said their insurance provider, HealthPartners, has sent three separate letters declining to cover this one-time $3.2 million treatment.

A spokesperson for HealthPartners told FOX 9 in a statement, "We’re not able to share specific information about this member’s situation or experience because of health privacy rules."

Rare DMD mutation

Liam has a rarer genetic mutation than most people who have Duchenne, and the denial letters from HealthPartners cite this as a reason Elevidys isn't a viable treatment option.

However, Dr. Eskuri said he meets all the eligibility criteria: He has Duchenne, he’s the right age, he’s walking, he doesn’t have a strong antibody reaction to the therapy, and he doesn’t have the genetic change that’s contraindicated by the FDA.

"I would be tickled pink if he got the drug tomorrow and flash forward 10, 20, hopefully, 30 more years from now and he's still in a spot where hopefully he can be walking independently and if not independently, maybe with minimal support," Eskuri said.

Gillette Children’s treats between 120 and 150 patients with either Duchenne or Becker Muscular Dystrophy, and only five patients total were deemed potential candidates for Elevidys due to their age. Two of those five have since been deemed ineligible. That leaves Liam, one patient who received Elevidys, and another who is still undergoing testing, according to Eskuri.

"About a third of our patients right now are not even being diagnosed until after they've lost eligibility for Elevidys. They've been diagnosed after their sixth birthday," she said.

That means time is of the essence.

"For the betterment of Liam and other children out there, we just need to get these kids treated as quickly as we can so that we can save their muscles."

The Schroeders said the next step is to have an independent third-party review of their case. They also created a change.org petition to raise awareness.

"We started this petition to hopefully get HealthPartners to understand that this is not OK. It's not OK to punish a 4-year-old for having a more rare genetic mutation of a specific disease," Joan Schroeder said. "We're just trying to get whatever we can for our son so that he can live the best life that he is capable of living, and I think we can do that with Elevidys."