Stifling regulations have turned treatment for terminally ill Americans into a bureaucratic nightmare. Last month, while many veterans celebrated Joe Rogan and President Donald Trump’s support for psychedelic drugs, those in the Huntington’s disease community faced another disappointment: UniQure, a company advancing a promising treatment, may be abandoning the U.S. market due to bureaucratic roadblocks.
I am not the president or the world’s most popular podcaster. I am a daughter who has tested positive for the Huntington’s disease gene and will one day exhibit the same symptoms that consumed my father’s personality and mind until he took his own life.
The FDA consistently responds to rare disease treatments with the same refrain: “Wait, wait, and then wait some more.”
I have advocated for the Huntington’s disease community in memory of my father and with hope for a different future. The outlook is grim for those like me unless the Food & Drug Administration permits access to treatments such as AMT-130, which UniQure is now advancing first in the U.K. after the FDA pushed the United States down the priority list due to unreasonable demands.
Those demands are disastrous for Huntington’s disease patients. Under the FDA’s proposed criteria, launching a placebo trial would require non-therapeutic brain injections—a practice that clashes with medical ethics.
Even without the ethical concerns of such trials, Huntington’s patients cannot afford the years it would take to complete them. We live on a timeline defined by a relentlessly progressive and fatal disease.
While I am glad veterans are receiving attention and influencers like Rogan have support, it raises a critical question: Why must it take a celebrity and the president to push the FDA to follow basic medical best practices?
For years, the rare-disease community has organized and advocated for change while managing devastating diagnoses. Parents of children with Duchenne muscular dystrophy and Sanfilippo syndrome have fought tirelessly as their children decline.
The FDA’s pattern remains unchanged: Wait, wait, then wait some more. This means families are running out of time on a clock that ticks louder with each passing month. We cannot afford years of unnecessary testing.
Rogan’s recent intervention shows the system can move quickly when it wants to—especially when voices with direct access amplify a cause. Now we need the same urgency applied to rare disease treatments.
Families like mine are not seeking special treatment. We simply want the choice to try new medicines when all existing options have failed. We know what lies ahead.
President Trump already made progress with the Right to Try Act, which provides terminally ill patients a pathway to access potentially life-extending treatments. It is critical that he push FDA officials to commit to the same right-to-try principles championed in his first term.
Scientists are making remarkable strides in rare disease treatments—but innovation matters only if patients can access them. Adults like me and children with terminal conditions whose parents approve are willing to accept any risk associated with new therapies.
Until someone steps up to advocate for people like me, our only alternative is the certainty of an illness that will slowly erode lives and then end them.
Rachel Reising is a Huntington’s disease advocate and nutritional therapy student who has received an HD gene positive diagnosis.
