Recently, while some veterans were celebrating Joe Rogan and President Trump, communities affected by Huntington’s disease faced another setback. UniQure, a company that seemed to offer hope with a potential treatment, may abandon the U.S. market due to regulatory issues.
I’m neither a renowned podcaster nor a president. I’m a daughter diagnosed with Huntington’s, a disease that one day will take over my life just like it did my father’s, leading to a tragic end.
When it comes to rare diseases, the FDA’s response often feels monotonous—always the same refrain: wait and wait some more.
In memory of my dad, I have fought for the Huntington’s community, hoping for a brighter future for myself. Without the FDA allowing access to treatments like AMT-130, concerns for those of us with Huntington’s remain dire. Currently, UniQure is prioritizing its treatment in the UK after the U.S. got stalled, tangled up in what seem like unreasonable FDA demands.
These bureaucratic demands are crushing for Huntington’s patients. Launching a placebo trial under these new FDA guidelines would involve giving non-therapeutic injections into the brains of participants, a practically impossible task. How does that align with medical ethics?
But beyond the ethics, we simply can’t afford to spend years in trials. We exist on a much tighter timeline, defined by a relentless and deadly disease.
While it’s great that veterans are receiving recognition and support from figures like Rogan, it brings up troubling questions. Why must celebrities and political leaders push the FDA to act on basic medical principles?
For years, the rare disease community has done everything asked of us to effect change. We organized, advocated, and worked tirelessly—even while dealing with devastating diagnoses and declining health.
Parents of children with conditions like Duchenne muscular dystrophy or Sanfilippo syndrome fight for their rights while watching their loved ones suffer.
Again, with rare diseases, the FDA’s advice tends to echo: wait, wait, and wait some more. The clock ticks ominously, as each month passes. We don’t have the luxury of time for unnecessary testing.
Rogan’s involvement shows that when there’s enough noise, the system can react quickly. It’s clear that when influential voices resonate, the president pays attention. We need to apply that same urgency to the treatment of rare diseases.
Families like mine aren’t looking for special favors. We just want the option to take calculated risks by trying new medications when all else has failed. After all, we already know the fate that looms over us.
Trump has made strides by supporting the right to try, which allows terminally ill individuals access to potentially lifesaving treatments. It’s essential that he continues to advocate for this principle with the FDA.
Scientific advancements in rare disease treatments are promising. But that progress means little if patients aren’t permitted to try these developed therapies. Adults like me and children facing terminal conditions, with parental consent, are eager to take on the risks of new treatments.
Until someone else voices these concerns, our only option feels painfully certain—a disease that methodically steals our lives before finally taking our breath away.
