Laura and Jordan McLinn
Laura McLinn of Indianapolis, Indiana, takes a selfie with her son, Jordan, 12, who has Duchenne muscular dystrophy (DMD). Credit: Laura McLinn

NASHVILLE, Tennessee—It’s been nearly 4 years since Jordan McLinn, who has Duchenne muscular dystrophy (DMD), tried twice to embrace then-President Trump at the White House signing of the controversial “Right to Try” bill into law. A video of Trump hugging the boy from Indianapolis, Indiana, and kissing him on the forehead went viral, and Jordan became a celebrity in the rare disease world.

But Jordan—now almost 13—never needed the legislation to access unapproved therapies, which is the focus of the Right to Try movement.

He has just marked 5 years of taking NS Pharma’s viltolarsen (Viltepso®), as one of only 16 boys in the company’s phase 2 trial for the once-a-week infusion, which is aimed at slowing the progression of Duchenne among the 8% of DMD patients who are amenable to exon 53 skipping.

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“He’s an amazing kiddo,” said his mother, Laura McLinn, during an interview at the Muscular Dystrophy Association’s 2022 Clinical & Scientific Conference here. “We’re here at this place, and he’s walking with me and attending all the sessions. He’s very happy. He’s living life, and he’s doing well.”

She recalls hearing about exon-skipping DMD treatments that were in the pipeline when Jordan was 5. “But at the time, from what we were hearing, he wasn’t going to meet the inclusion criteria to make it into the clinical trial because of his age,” she said.

That led her to start lobbying heavily for Right to Try legislation, which offers a pathway for patients with life-threatening illnesses to access treatments to which they would otherwise not have access. A number of patient advocacy groups supported Right to Try, though the concept was opposed by the National Organization for Rare Disorders (NORD) and the US Food and Drug Administration (FDA) on the grounds that unapproved treatments could cause rare disease patients more harm than good.

“When we first worked on Right to Try, it was at the state level in Indiana when Mike Pence was our governor. At the time, he fell in love with Jordan. They became best buddies. He said, ‘if you ever need anything in the future, let me know,’” McLinn said.

“Little did we know that Mike Pence would later become vice president of the United States,” she said. “Jordan and I started working on federal legislation. To be clear, by that time, Jordan had already made it into a clinical trial. It was no longer so personal to us, but we had met so many patients along the way that weren’t as lucky.”

For more than 3 years, the McLinns would drive round-trip to Chicago, Illinois, every week for the boy’s infusion. During the same period, they also made 30 to 40 trips to Washington to lobby for Right to Try.

But eventually, the FDA granted accelerated approval for viltolarsen, so access is now available commercially. A nurse comes to Jordan’s home in Indianapolis and gives him the drug intravenously; in February 2022, the boy marked 5 years on the treatment, which doesn’t cure DMD but slows the progression of the disease by increasing dystrophin levels.

Indiana State Capitol in Indianapolis. In 2015, Indiana signed Right to Try legislation into law; 3 years later, the federal government followed suit. Credit: Larry Luxner

“To see all that hard work come full circle was incredible,” she said. “Since then, I’ve had many individual families reach out to me to share their story and to thank Jordan and I for involvement with Right to Try. I said all along that if Right to Try can help one patient, to me it’s worth it. I know not everyone would agree with that, but that’s OK.”

As governor, Pence signed the Right to Try legislation into Indiana law in 2015. Three years later, Trump signed similar legislation into law at the federal level—and the McLinn family was there for the ceremony, along with amyotrophic lateral sclerosis (ALS) patient Matt Bellina and others who had fought for the cause.

“To this day, I still ask myself the question, ‘Why would anyone oppose this?’ To me, it was a no-brainer,” McLinn said. “There’s a lot of ethics around it, but for me, I’m very passionate about making sure that, no matter what the pathway is—whether it’s a clinical trial, an approved treatment, or expanded access—every individual has the right to try to pursue that, in whatever way makes the most sense for them.”

McLinn is also a small business owner and founder of the nonprofit Best Day Ever Foundation. The organization describes itself as a “one-stop shop” for Indiana families affected by Duchenne.

For the past few years, McLinn has been hosting monthly “Living our best lives with Duchenne” meetings via Zoom, in which she shares what all other DMD organizations are doing. In June, the group plans to begin face-to-face meetings.

“We inform families of every event and every resource out there,” she said, noting that Indiana is one of only a handful of states where families can qualify for Medicaid waivers as soon as their sons are diagnosed with Duchenne—even as early as age 3 or 4—regardless of income.

“Medicaid waivers are unique to every state, and for some states, the waitlist is literally years, and our boys can’t wait years,” she said. “We’re very lucky because some states don’t have that. In Indiana, you get money you can use for respite care and therapies. During COVID, I was able to help more than 20 moms quit their jobs, stay home and care for their child full-time, and get paid through the Medicaid waiver to be able to do that. It has been life-changing for many of our families. Now I’m trying to teach parents to mentor other parents.”