When my husband Dave and I learned that two of our three daughters had a fatal genetic disease, we entered the kind of nightmare for which no parent can fully prepare. Both girls — Keira and Livvy — were diagnosed with metachromatic leukodystrophy, or MLD, a rare degenerative disorder that destroys the nervous system and gradually robs children of the ability to walk, speak, swallow, and ultimately survive.
But Keira had something that Livvy did not: time.
Keira’s disease was identified early enough for her to qualify for an experimental gene therapy that was unavailable in the United States. To save her life, our family relocated to Italy for five months and raised roughly $500,000 so she could receive treatment. Keira became the 32nd child in the world to undergo the therapy. Today, she is a thriving 6-year-old with zero symptoms — running, laughing, attending school, and living the kind of childhood we once feared she might never have.
Livvy was not as fortunate. By the time doctors diagnosed her, the disease had already progressed too far for the treatment to work. While we were fighting overseas to save one daughter, we were also forced to watch our other daughter quickly deteriorate. In just three months, she lost the ability to walk, then talk, then became tube-fed. Now she is in hospice at age 8, taking 11 medications a day to stay pain-free.
No American family should have to watch the clock run out while bureaucracy decides whether they are allowed to fight for their child’s life. Nor should they have to travel overseas to access a lifesaving treatment.
Our experience is why Congress should update the federal Right to Try law by passing the Right to Try for Individualized Treatments Act, also known as Right to Try 2.0, introduced by Sen. Ron Johnson (R-WI) and Rep. Diana Harshbarger (R-TN).
We are living through a new era of medicine, one defined by gene therapies and precision medicine tailored to rare diseases and unique genetic mutations. Yet America’s regulatory system remains rooted in the past, built around broad patient populations, lengthy approval timelines, and standardized commercial pathways.
For those facing fast-moving diseases, time matters. When a child, like our daughter Livvy, is losing abilities week by week, waiting years for approvals may mean the treatment comes too late. For our family, it meant the difference between a child in hospice and a child in the classroom.
The original Right to Try law was created to give patients with life-threatening illnesses a chance to access promising treatments when no other options remain. Right to Try 2.0 builds on that idea for a new era of personalized medicine.
The bill would allow eligible patients, working with their doctors, to pursue individualized investigational treatments while still maintaining important patient protections and informed consent requirements.
It does not guarantee a cure or force companies or insurers to provide treatment. It simply protects a family’s right to try when time is running out.
That is all my family wanted. We weren’t demanding a guaranteed cure. We simply wanted the chance to pursue one before the window closed.
RIGHT TO TRY: TRUMP’S DECISIVENESS CAN SAVE CHILDREN WITH RARE DISEASES
Keira is alive today because we refused to accept waiting as the only answer. Livvy’s journey, meanwhile, shows the devastating cost of delay. The problem was not that science had no answer. It was that our regulatory system made it difficult for us to reach it in time. The law kept families like ours from reaching it in time. Congress has the power to change that and should move swiftly to pass Sen. Johnson and Rep. Harshbarger’s Right to Try for Individualized Treatments Act.
By doing so, lawmakers can bring the U.S.’s regulatory framework into the age of gene therapy while honoring a fundamental principle: In a country that leads the world in medical innovation, no family should have to travel to another continent for the chance to save their child’s life.
Kendra Riley is a business owner and mom of three girls, two of whom were diagnosed with MLD in 2020. She has since become an advocate to other rare-disease families like hers across the nation.