There’s nothing quite like a “buzzer-beater” shot that wins the last game at literally the last second. Everyone loves to see a desperate, half-court shot that turns a loss into a victory, whether it’s the NBA or a high school game. Players on a losing side have nothing to lose, and sharing their long-shot glory makes us all feel like we have more control over our lives.
Of course, sometimes people who need to beat the buzzer have much more at stake than a basketball game. There are many families worldwide suffering from rare diseases who are hoping that a proverbial buzzer-beater could save lives.
Over the last year and a half, two parallel stories have emerged that reveal a critical gap in how governments prioritize access to rare disease treatments, one in the nation of Georgia across the world and the other here in Washington, D.C.
In Georgia, families of children with Duchenne Muscular Dystrophy (DMD) have maintained a continuous protest for more than 500 days, sleeping on wooden benches outside the government building while their children deteriorate. About 100 children in Georgia have DMD; three have died during the course of this protest. Because it’s so rare, treatment doesn’t get a lot of resources. Meanwhile, here in the United States, rare disease advocates staged a mock funeral at the offices of the Food and Drug Administration (FDA) in March 2026 – complete with caskets and photos of dying children.
This is part of the two-edged sword of modern technology. On the one hand, advanced medicine promises to save and improve lives in ways never before dreamed – on the other hand, it’s difficult for traditional institutions to keep up.
During President Trump’s first term, he signed right-to-try legislation to try to enable experimental research for seriously ill patients. The idea is if standard treatments have failed, patients can go beyond the standards set by the FDA for experimental therapies that have not yet been approved for public use.
It’s not an ideal situation – but neither is letting a child die of muscular dystrophy or something equally horrible. Those patients and families have nothing left to lose. However, the FDA has proven unwilling to give up its control regarding medical approvals. The agency’s foot-dragging drew the attention of The Wall Street Journal last year, reporting that “In recent weeks, the FDA has rejected three therapies for debilitating diseases that have shown promise in clinical trials. The agency has also forced off the market a gene therapy that can slow the degenerative loss of muscular function in young boys with certain genetic mutations.”
These protests are not isolated incidents. They’re warning signs of what happens when institutional channels fail, and desperation reaches a breaking point. And while one hopes that the institutions of our republic are sound, the Georgian protests could reveal the trajectory that American advocacy could follow if the FDA does not fundamentally change its approach to rare disease approvals.
Many leaders want to help desperate families make a full-court press. Sen. Ron Johnson (R-WI) announced an investigation into the FDA’s bizarre denials of right-to-try research; FDA countered that approvals are "at their peak" under Commissioner Marty Makary.
Makary has a history of breaking promises, purging senior leadership, and sowing fear throughout the FDA. He has created a regulatory culture that seems more focused on infighting than saving lives – inexcusable when kids’ lives are on the line.
For families both here and in Georgia, underlying frustration has been building slowly – but accelerating for kids who are working against a ticking clock. In both situations, families have to watch their children suffer and possibly even die while regulatory agencies are doing nothing to help get the ball up the court.
The FDA's rejection of multiple rare disease treatments in early 2026 (RGX-121, drugs from Biohaven, Saol Therapeutics) has created a "communications crisis" at the agency that's generating escalating frustration. Meanwhile, in Georgia, the government has pushed back on the protests, which seems bizarre for something as innocuous as sick kids.
That hasn’t happened here, but the FDA has publicly attacked biotech firms, like uniQure, in March, creating a hostile dynamic when cooperation seems more appropriate.
The time for FDA reform is not measured in months, but in the remaining lifespan of children who see time running out on the clock. The question is not whether American protests will intensify, but whether the agency will act before they do. There’s no reason for the FDA to try to block shots from doctors and families when it should be trying to help them beat the buzzer.
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