Be Louder Than the Chaos: Why Now Is Not the Time to Pause

There is a silence creeping in—one that masquerades as strategy, patience, or professionalism.

But let’s be honest: it’s paralysis. It's exhaustion. It’s fear disguised as “waiting for the right moment.”

And while we wait, children are still being denied formula. Therapies are still capped. Newborn screenings are still delayed. And the FDA is still rejecting rare disease treatments over paperwork instead of patients.

The truth? You should be madder than ever. And you should fight harder than ever.

Because in the face of political chaos—budget cuts, agency shakeups, and leadership that doesn’t understand rare disease—silence is complicity.

You’re Not Imagining It—It’s Getting Worse

This isn’t drama. It’s documented failure.

Rare disease families are facing denials, delays, and systemic neglect. We see it in the PICU stays that follow late diagnoses. We see it in children who still seize, who can’t speak, who are turned away from therapies they desperately need.

We see it in drug rejections like govorestat—a therapy that showed promise in reducing tremors and improving speech, but was tossed aside because of missing data from a handful of patients in a tiny trial. The FDA calls it regulatory rigor. We call it a missed opportunity. A life lost to red tape.

And still, the response from too many is: Let’s wait. Let’s see what happens after the election. Let’s not ruffle feathers.

No.

Stop Waiting. Start Roaring.

We are in a moment that demands louder voices—not safer ones.

There’s a new administration. Agencies like CMS and NIH are under pressure. Approvals are slowing. Funding is uncertain. But the needs haven’t paused—so neither should we.

Rare disease parents don’t have time for political etiquette. We don’t have the luxury of pause.

We have children whose bodies produce galactose internally—even with strict dietary treatment. That galactose has nowhere to go and builds up in tissues like the brain, eyes, and ovaries. This causes real harm—cognitive delays, tremors, and infertility that no label can erase.

If that doesn’t demand fury—what does?

This Isn’t Partisan. It’s Personal.

This isn’t about red or blue. It’s about right and wrong.

It’s wrong that CMS still denies medical formula unless your child is tube-fed. It’s wrong that therapies with clear clinical need are capped at 20 visits. It’s wrong that SSA doesn’t recognize galactosemia as a disability despite clear evidence of lifelong challenges.

And it’s beyond wrong that the FDA can see promise in a drug trial, hear from families and clinicians alike, and still reject the treatment—because it doesn’t fit a mold that was never built for rare disease.

So yes, you should be mad.

You should be infuriated. And you should channel that rage where it belongs: into advocacy.

The Only Way Forward Is Through

Here’s what you do:

• Write Congress. Demand action on H.R. 6892, the Medical Nutrition Equity Act.

• Email the FDA. Push for rare disease flexibility and reconsideration of govorestat.

• Call CMS. Insist on equity in therapy and formula coverage.

• Make noise. On social media. In your statehouse. At your next IEP meeting.

There are only 3,500 of us in the galactosemia community—but we are not alone. We stand among 30 million Americans with rare diseases. This is not small. This is not niche. This is national.

We don’t need pity.
We need policy.
We need to be louder than the chaos.

If our leaders won’t rise to the moment, we will.