By: Sherine Blackford
My son Loic had the kind of life every parent hopes for – school, playing with his sister, and enjoying life like other children. He was diagnosed with PKAN at just 2 ½ years old. Although he had development delays, he continued to make progress after his diagnosis.
Then, beginning in October 2025, over the course of about 8 weeks, this rare neurological disease called Pantothenate kinase-associated neurodegeneration, or PKAN for short, took away his ability to walk and stole the other abilities he had worked so hard to gain. There was no warning or any chance to prepare. We just watched as this cruel disease rewrote our lives in a matter of weeks.
There is no treatment or cure for PKAN. Families like ours can’t afford to simply wait and hope. We have traveled across the country to see specialists, worked alongside researches, and founded the Loving Loic Foundation, which is actively funding and advancing the development of a gene therapy for PKAN. Every day, we are fighting to create the answer our children need. But the sad truth is that PKAN is always going to win until we have an effective treatment.
When my son was first diagnosed, doctors told me there was hope that clinical trials were coming, that researchers were asking the right questions, and that the scientific community hadn’t given up. I held onto every word of that.
You see, for families like mine, the research pipeline is the difference between a life sentence and a fighting chance, and so, every dollar invested in that research matters. That’s why when I see Congress considering policies that may affect investment in biomedical research, my first thought is about Loic and every other family still waiting for an answer.
As I understand it, the Most Favored Nation plan Congress is debating would tie the prices paid for certain drugs to prices in other countries. Many people have raised concerns that changes to drug pricing could also affect investment in research and development, particularly for rare diseases where bringing a therapy to market is already extraordinarily difficult. While I recognize there are legitimate concerns about the affordability of prescription drugs, I also hope policymakers carefully consider how any reforms could affect innovation for patients with rare diseases like PKAN.
As Congress considers different approaches, we need to ensure that the research environment that has helped so many families stays intact. I urge policymakers to think carefully about what gets sacrificed when we change the economics of drug development.
PKAN is always going to win until we find an answer. I have accepted that this disease is relentless. What I refuse to accept is that we stop looking for one.
