Bonus: The Path with Becky Quick: A Father’s Rare Determination 7/2/26

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**John Crowley** (1:04)
Thank you.

**Becky Quick** (1:05)
Thank you, and we'll see you next time. Thank you.

**John Crowley** (1:10)
Desperation, fear, determination.

**Becky Quick** (1:19)
I'm Becky Quick. Welcome to The Path from CNBC Cures, a podcast series about the people, the struggles, the science of rare disease, and the determination to do something about it. I'm a mom of a child with a rare genetic disease.
My nine-year-old daughter, Kaylee, has a condition that's called SYNGAP1. She's part of a small patient group. There are only about 1,700 other people in the world with this diagnosis.
Essentially, she only produces half of the SYNGAP protein that's needed for brain development. That means that she has seizures, developmental delays, and intellectual disabilities. But what Kaylee doesn't lack is courage. Go.

**SPEAKER_1** (2:11)
Or determination.

**Becky Quick** (2:13)
Recently, Kaylee went a little further into the world with a little less of a safety net.

**SPEAKER_6** (2:18)
Swim, baby.

**Becky Quick** (2:20)
Swimming on her own at our local pool without a swim belt for the very first time.

**SPEAKER_6** (2:25)
Kaylee, you can do it.

**Becky Quick** (2:27)
She did great. She went for it. She jumped right in, her feet touched the bottom, she popped right back up. Kaylee, I'm so proud of you. Then she swam from one end of the pool to the other.

**SPEAKER_7** (2:38)
Yay for Kaylee.

**Becky Quick** (2:40)
On her own power, determined to do it on her own. That moment was a breakthrough for Kaylee, but it took so much work for her to get there.

**SPEAKER_8** (2:50)
No problemo.

**Becky Quick** (2:56)
We were, we are, so proud.

**SPEAKER_9** (3:00)
Ready? Three, two, one, we're not waiting.

**Becky Quick** (3:05)
My guest today is the father of not one, but two children with a rare disease. He knows a lot about transformations that seem sudden, but have extraordinary work and raw courage behind them. John Crowley took the shocking news that his two small children, one just a newborn, had fatal diseases. He changed his entire life and he made a breakthrough so incredible that it became a Hollywood movie.
And what was amazing to me was that I had met John Crowley 10 years ago. And welcome back to Squawk Box here on CNBC, first in business worldwide.

**SPEAKER_8** (3:42)
Now let's bring in John Crowley. He's the CEO of Amicus Therapeutics, which focuses on rare diseases. John, it's good to see you on set.

**John Crowley** (3:49)
Great, great to be here.

**Becky Quick** (3:50)
He was a guest on Squawk Box in the summer of 2016
I was there, I was pregnant with Kaylee, and I had no idea that I was about to join the rare disease path that John had been on for so long. John, first of all, what is Pompe disease and how are your kids?

**John Crowley** (4:06)
Sure, so Pompe disease is a rare neuromuscular disease.

**Becky Quick** (4:10)
In fact, I had no memory of that segment of even meeting him until it was pointed out to me while we were planning for this interview. What you're talking about is incredibly noble.
I laud it, but what generally happens with smaller companies is that, you know, one of these big pharmaceutical companies is going to wait until you have a hit and then try and buy the company. Your market capitalization is under a billion dollars. How do you prevent that from happening?

**John Crowley** (4:31)
Keep focusing on building an independent, fully integrated biotech company where, you're right, we're in that unique space now, Becky, where we have an approved drug, but we're also working on a terrific pipeline.

**Becky Quick** (4:44)
Tells you a little bit about how life can change and how quickly. Good?
Is that out of your shot? Do you guys need the?

**John Crowley** (4:54)
I'll do it for you. Take one.