RIGHT TO TRY: TRUMP’S DECISIVENESS CAN SAVE CHILDREN WITH RARE DISEASES

The rare disease community and people like me want to make sure senators demand fiscal accountability from the FDA to ensure it uses these resources for its stated mission.

I lost my grandmother and my father to Huntington’s disease, an incurable degenerative neurological disorder that slowly destroys the body and mind.

Seven years after my grandmother died, my father decided to end his own life rather than continuing to cognitively decline and face losing the ability to talk, walk, swallow, and feed himself — the same problems he watched his own mother suffer. He knew he could not live anything approaching a normal existence.

When I found out that I carried the same gene for Huntington’s disease, I decided I wasn’t going to sit back and wait for the inevitable. I was going to fight to help everyone else experiencing the same bitter struggle. 

I am not a scientist, politician, or healthcare executive. I’m a normal American hoping for a better outcome than my father faced.

Across the globe, researchers are doing extraordinary work on diseases once considered hopeless, developing therapies for Huntington’s disease and many other devastating conditions.

That should give families hope. The problem is that even when breakthroughs happen, there is no guarantee the FDA will allow people to access treatments quickly enough to make a difference. Families spend years trapped in the purgatory of regulatory delays, such as the one in March, when the FDA declared that the Huntington’s disease treatment AMT-130’s promising results weren’t good enough, despite what one expert called “no documented rationale.”

Weeks later, the U.S. company that makes AMT-130 announced that because of the FDA blockade, it was going to release the drug — not in the United States, as I had hoped, but in the U.K. While FDA bureaucrats dither, patients lose abilities they will never regain.

The Senate hearing scheduled for May 13 was postponed due to the last-minute resignation of now-former FDA Commissioner Dr. Martin Makary. This delay can provide Congress with a renewed opportunity to fully understand what is at stake in the FDA budget. For rare disease patients, nearly 30 million U.S. adults, and half of us children, it’s not just numbers.

FDA decisions shape the pace of scientific review, access to treatment, and the future of American medical innovation. Patients facing terminal conditions cannot afford unnecessary regulatory paralysis.

Whomever President Donald Trump appoints as the next FDA commissioner must ensure that patients can make informed choices about experimental treatments when existing options have failed. Questions about past resource allocation also must be answered.

Trump took an important step by signing the Right to Try Act, creating a pathway for terminally ill patients to access experimental therapies. That principle matters because when you know that a disease will eventually kill you or a loved one, the risk of trying a promising treatment is far preferable to the risk of doing nothing. 

So many families fighting conditions such as Huntington’s, Duchenne muscular dystrophy, and Sanfilippo syndrome traveled to Washington this week, at great personal cost, to express this important truth to our leaders.

My hope is that Congress and the administration will hear us. Rare disease patients should not have to slowly waste away without being able to use the drugs our own scientists invent. 

TRUMP TO FIRE MARTY MAKARY? IT’S ABOUT TIME FOR CHANGE AT FDA

Folks like me may not have celebrity status, political influence, or millions of social media followers. We are just people facing some of the most devastating conditions imaginable, and we deserve the chance to fight for our futures with innovative treatments before it’s too late. 

We need to be heard — because we’re running out of time. And some of us will run no further.

Rachel Reising is a Huntington’s Disease advocate who has received an HD gene-positive diagnosis.