The Impossible Trade-Off

by Matthew Might, PhD

Ashley and I have known each other since college, but it is the shared journey as special needs parents that has kept us in touch over the years.

When my son Bertrand passed in 2020, she reached out with an authentic and rare compassion.

When I saw that her son Declan had passed recently, I reached out, hoping I might be able to offer the same in return.

We talked about some things that only special needs parents can really understand—the hard things, the complex grief, the impossible trade-offs.

I'd like to talk about one of the trade-offs that almost never gets mentioned publicly.

Stated plainly: Do you spend more time with your child? Or do you take time away from your child to push for treatments, for community, for cures—through science, through advocacy, through whatever means you have?

To be honest, I don't think there's a way to answer this question without feeling like you're losing.

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My son Bertrand had NGLY1 deficiency. He was the first person in the world diagnosed with it.

For nearly thirteen years, I watched him fight his body while his spirit stayed gentle and joyful. And for nearly thirteen years, I faced that question every day.

The math is brutal and cruel.

If your child has any one of the many thousands of ultra-rare genetic disorders, a cold, honest assessment of the odds of saving their life even with maximum advocacy places it close to zero.

The science often doesn't exist yet.

The treatments might be decades away. The funding isn't there. The patient population is too small for pharma to care.

So, the "rational" thing to do might seem obvious: spend every possible moment with your child.

Don't tilt at windmills.

Don't fight to build a community.

Don't disappear into a lab.

And, don't spend time on Capitol Hill when your kid needs you at home.

But there's the problem with that logic.

If every parent makes that choice—the rational choice, the entirely understandable choice—then there is no forward progress.

No new science.

No new legislation.

No new patient communities forming.

No one pushing back against the void.

And if no one pushes, then there's truly zero chance for all -- not just for your child, but for every child who comes after.

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Progress, it turns out, depends on parents making an irrational sacrifice: giving up some of the precious, scarce time they will have with their child, in exchange for odds that remain the best approximation of zero anyone can encounter.

Lottery winners face easier odds.

It already takes winning one lottery to even become a second special needs parent.

To believe you can race to a treatment in time is to think you can win the lottery again.

Even so, I bought those lotto tickets.

I tried to save Bertrand's life.

Something in me couldn't rest at night if I knew there might be a chance at saving his life. Any chance.

Instead of spending every moment with Bertrand, I spent nights writing code when I could have been reading to him.

I went to DC when I could have been home.

I wrote papers and chased grants and raised funds.

I told myself it was for him.

And it was.

And, it was also for me: so that I could sleep at night without that feeling of giving up on him robbing me of sleep and sanity.

But, it was also time I didn't get back once he was gone.

In the hours before he passed, a dear friend called and these words fell out of my mouth:

"I'm afraid that I have run out of time to do science."

It was impossible not to feel in that moment that I had made the worst of all possible choices with my time.

Sometimes, the things I tried worked.

Bertrand got treatments that helped him.

He cried real tears for the first time at age six because of a compound we identified.

His seizures improved.

Science I helped push forward is now helping other patients, including patients who are still alive today because of work that Bertrand's life made possible.

But, sometimes, it didn't work.

Sometimes I was just gone.

And then Bertrand was gone.

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In the end, after much grief, regret and self-judgment, here's what I've learned: there was never actually a wrong answer.

This is not a test.

There is no grade.

There is no "correct" path that, if you'd only found it, would have saved your child and preserved every moment.

Some parents pour everything into presence.

They are there for every seizure, every feeding, every quiet moment.

That is love. That is valid. That is enough.

Some parents pour themselves into advocacy, into science, into fighting systems that don't want to be fought.

That is also love. That is also valid. That is also enough.

Most of us stumble somewhere in between, never sure we're getting it right.

And here's the part I wish someone had told me earlier: you will feel guilty no matter what you choose.

The more you stayed, the more you'll wonder if you should have fought harder.

The more you fought, the more you'll wonder if you should have stayed longer.

This guilt is not evidence that you chose wrong.

It is simply the best a broken human heart can do when trying to process what it is like to have loved in the face of impossibility.

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Ashley wrote about the relief she felt after Declan passed—and the difficult emotions that come with expressing this.

I understand that, too.

There's a version of this same honesty I want to offer about the guilt.

After Bertrand died, I replayed decisions.

The trips I took. The papers I wrote. The time I spent in meetings instead of with him.

And what I've come to accept is this: I did the best I could, given what I knew, given who I am, given the impossible situation I was in.

So did his mother. So did everyone who loved him.

You cannot judge your past self with the knowledge of your present self. That's not fairness. That's cruelty.

If you made the best choice you could with the information you had in the moment, then you did the best you could. 

When the uncertainties are resolved later—when you learn what you couldn't have known then—it doesn't change the fact that you did your best at the time.

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There's something else I want to say, though.

Something that has only become clearer in recent years.

The "finish line" I dreamed about when Bertrand was young is getting closer.

In 2018, a six-year-old girl named Mila Makovec became the first person in history to receive a drug created just for her.

Her mother, Julia Vitarello, had pushed researchers at Boston Children's Hospital to design an antisense oligonucleotide—a personalized genetic treatment—for Mila's unique mutation causing Batten disease.

They went from diagnosis to treatment in less than a year. That had never been done before.

Mila passed away in February 2021—the disease had progressed too far by the time the drug arrived—but her life proved that crossing the finish line within a single child's lifetime had become possible.

It was the first "n of 1" drug, and they named it milasen.

In October 2023, almost exactly 3 years after Bertrand passed away, Matt Wilsey, another NGLY1 dad who had been tilting at windmills with unrelenting intensity texted me.

He had slayed the dragon.  He crossed the finish line.

A trial for a gene therapy for NGLY1 was a go.

A few months later, the first patient received gene therapy for NGLY1 deficiency—Bertrand's disease.

Then, in February 2025, a baby named KJ became the first person to receive a personalized CRISPR gene-editing therapy.

He was born with a severe metabolic disorder called CPS1 deficiency that would have killed him in months.

A team at Children's Hospital of Philadelphia designed, manufactured, and received FDA approval for a bespoke treatment in just six months.

KJ is now home, thriving.

At a recent conference in Boston, I met baby KJ in person.

Chubby and delightful, I could see Bertrand smiling back.

And then there's Amber Freed.

When her son Maxwell was diagnosed with SLC6A1 in 2018—a condition so rare it didn't even have a name—she was told to "go home and give him the best life you can."

Instead, she quit her job, taught herself molecular biology, tracked down scientists, raised millions of dollars, commissioned the creation of mouse models with Maxwell's exact mutation, and essentially willed a gene therapy into existence.

In September 2025, Maxwell became the first person to receive treatment for his disease.

Amber earned an "honorary PhD" through sheer determination and love. (She described giving herself one after reading "every textbook and journal article imaginable," starting with Bioinformatics for Dummies.)

In a recent interview, Amber stated the no-win nature of the choice parents face with perfect clarity: "I've spent all of this time fighting for him and not spending time with him. That came at an incredible cost for myself and him. But what if it's the wrong decision and it's not gonna work? But I can say in my gut that I knew as a mom that this was his one chance to live."

None of these breakthroughs happened in a vacuum.

Milasen built on the science and pathways carved out by parents who came before, but not quite far enough or fast enough.

The NGLY1 gene therapy emerged from a patient community that formed because Bertrand's story went viral and connected families who didn't know they existed.

Baby KJ's treatment relied on CRISPR tools refined over years by researchers funded by patient foundations.

Maxwell's gene therapy happened because Amber spent seven years building an infrastructure—scientific, financial, political—that didn't exist when he was diagnosed.

Every one of these advances stands on the shoulders of parents who made the impossible choice. Parents who sacrificed time with their children to push science forward, even when the odds were near zero, and even when they knew they might not cross the finish line in time.

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With Bertrand gone, I find gratitude in the fact I don't have a choice to make anymore --  that I now have the freedom to spend all of my time dedicated to finding treatments, even if for others.

I'm beyond fortunate that my day job is to direct the Hugh Kaul Precision Medicine Institute at UAB, where over the last eight and a half years, I've been able to build a team and research infrastructure capable of helping hundreds of patients like Bertrand.

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So here's what I want to say to anyone else staring at this impossible trade-off:

Whatever you choose, choose it with as much intention as you can. And then forgive yourself for not being able to do the impossible.

If you spend every moment with your child, that is a gift.

If you spend some of that time fighting for a future that may never come, that is also a gift—maybe not to your child, but to children you'll never meet.

And if you do some chaotic, imperfect mix of both, stumbling through it like the rest of us?

That's not failure. That's love, doing the best it can in a world that was never designed for this.

Bertrand taught me that acceptance isn't surrender.

It's just acknowledging the world as it is, and then doing the best you can within it.

And doing the best you can is not only all you can do: it is enough.