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The Path with Becky Quick: Finding Hope 1/30/26 - Squawk Pod | Wave AI Podcast Notes

The Path with Becky Quick: Finding Hope 1/30/26

Fri Jan 30 2026

Luke Rosen was working as an actor and writer in New York when his daughter Susannah was diagnosed with KIF1A, an ultra-rare neurodegenerative condition. It's often fatal. At the time they received Susannah's diagnosis, Luke and his wife Sally didn't have much hope. There was no treatment for KIF1A, and there wasn't much work being done on it among researchers. But Luke, one of the most optimistic people you'll ever meet, did what so many parents of children with rare diseases do...he threw himself at the problem. He and Sally started the KIF1A.org Foundation so they could start building research that could go towards finding a treatment for their daughter. It was the beginning of a long journey for the family. Ultimately, that journey led them to the n-Lorem Foundation, an organization founded by CNBC Cures Advisory Board member Dr. Stan Crooke that offers free ASO treatments to individuals with nano-rare diseases. Thirty years in the making, Antisense oligonucleotide (ASO) te...

Summary

Squawk Pod: The Path with Becky Quick – “Finding Hope” (1/30/26)

Podcast: Squawk Pod
Host: Becky Quick (CNBC)
Guests: Luke Rosen (Father, rare disease advocate), Dr. Stan Crook (Founder, Ionis Pharmaceuticals & n-Lorem Foundation)
Date: January 30, 2026

Episode Overview

This episode of "The Path" dives deep into the world of rare diseases through the personal stories of families affected and the pioneering science that gives them hope. Becky Quick, whose own daughter lives with a rare genetic disorder, sits down with fellow parent-advocate Luke Rosen and Dr. Stan Crook, a trailblazing scientist developing personalized genetic therapies. Together, they discuss the emotional, medical, and societal challenges facing patients with ultra-rare conditions and the fight for treatments that can change lives.

Key Discussion Points & Insights

1. Personal Journeys of Rare Disease Parents

Sharing Difficult Stories
- Becky reflects on going public about her daughter Kaylee’s journey with Syngap1, a rare neurodevelopmental disorder. She underscores the importance—and personal difficulty—of sharing these private struggles for greater community awareness and support.
  - “We are both very private people and we are especially protective of Kaylee. And not being able to ask her consent on this project made us second guess ourselves for a long time.” (Becky Quick, [02:29])
Isolation and Finding Community
- Luke Rosen describes the shock of diagnosis for his daughter Susanna (who has a KIF1A mutation) and the rapid immersion into the rare disease world, where families bond over shared hardship and advocacy.
  - “You find your tribe quickly... You find somebody else who’s feeling as isolated as you are.” (Luke Rosen, [07:27])

2. Understanding Rare and “Nanorare” Diseases

Medical Explanation and Challenges
- Dr. Stan Crook introduces “nanorare”—diseases with fewer than 30 known patients globally—and explains how genetic sequencing is redefining what rare means.
  - “A nanorre mutation is a pathogenic mutation with a known prevalence of less than 30 humans in the world. If you can imagine the isolation... when you’re one of 30 or one of 10 patients in the whole world.” (Stan Crook, [17:11])
- For families, even basic information about conditions can be hard to access or afford.
  - “I couldn’t afford the $35 on PubMed... My God, everybody has the right to learn about their disease.” (Luke Rosen, [42:05])

3. The Science of Antisense Oligonucleotides (ASO)

Breakthrough Therapy
- Dr. Crook and Luke explain how ASO therapies—short strands of engineered genetic material—can “silence” or fix genetic errors. While not always curative, ASOs can dramatically improve symptoms.
  - “Instead of poly, many nucleotides, little oligo, a few of these genetic letters... bind to a specific target in RNA.” (Stan Crook, [21:22])
From Theory to Hope
- Luke’s journey to access ASO therapy for his daughter was arduous, involving advocacy, fundraising, and relentless outreach to companies like Ionis and scientists like Dr. Crook.
  - “That’s one of the first times I felt a lot of hope. On that island of rare disease... I felt other people coming onto it and saying, hey, do you need a raft to get off this island?” (Luke Rosen, [23:15])

4. Experiencing Treatment and its Impact

First Doses: Fear and Anticipation
- Luke recounts the emotional ordeal as Susanna receives her first treatments—hoping, waiting, fearing the worst, but persisting for even a small sign of improvement.
  - “If this is the last thing she sees... do I want her to know exactly how I feel and that I’m terrified and crying?” (Luke Rosen, [03:03]/[25:23])
  - “I did [smile], and I lied to her because I... forced that smile. Imagine how scared she was.” (Luke Rosen, [26:59])
Notable Improvements
- The first clear sign was extraordinarily simple yet profound: a peaceful family breakfast, free from Susanna’s tremors.
  - “It was the fact that it was quiet… her tremor was gone... That’s when I knew that the drug was working.” (Luke Rosen, [31:54])
- Other benefits included less pain, improved energy, and the ability to enjoy daily experiences as a family.
  - “If you can eliminate pain, like, God, that was. That’s it.” (Luke Rosen, [35:30])
- But challenges remain, including new symptoms like catatonia and the continued progression of disease.

5. Daily Struggles, Family, and Resilience

Emotional and Marital Toll
- Becky and Luke talk openly about the impact on their marriages—the constant vigilance, alternating care shifts, and the strain on a partnership.
  - “It’s so hard. I mean, we… can’t remember the last time Sally and I spent the same night in the bed. Nope.” (Luke Rosen, [42:38])
  - “That statistic that 80% of marriages fail in a rare disease community. And that doesn’t shock me because it is so hard…” (Luke Rosen, [43:12])
- The importance of support systems and letting go of outside judgment is emphasized.
  - “If everyone in the world just stopped caring about what people thought of them... The world would be better place, probably.” (Luke Rosen, [44:28])
Celebrating Small Victories & Holding on to Hope
- Even amidst relentless adversity, moments like singing the National Anthem or sharing a meal become shining milestones.
  - “She sang the National Anthem. She worked on for eight months... the kids were singing the National Anthem in their own way.” (Luke Rosen, [39:53])

6. Privilege, Access, and Advocacy

Both Becky and Luke acknowledge that their networks and resources give their children advantages many families lack—access to experts, information, and experimental therapies.
- “We've both met people along the way who are so much less fortunate, and those are the people that I would most like to help on this.” (Becky Quick, [40:42])

Notable Quotes & Memorable Moments

On Diagnosis and Grief
- “I thought in my head, maybe I could give Sally another two... minutes without knowing this, without knowing that our daughter is really sick…” (Luke Rosen, [10:11])
- “Another ignorant day. Instead we have to become smart in ways that we never thought we had to be.” (Luke Rosen, [12:00])
On Scientific Obstacles
- “Sickening to say the word market size in the same sentence as our kids’ horrific degenerative disease.” (Luke Rosen, [23:02])
- “Bad branding... as people have told me for about 40 years now.” (Stan Crook on ‘antisense oligonucleotide’, [22:21])
On Family & Hope
- “If we can slow that down and curb that severity of disease, then maybe... I’ll be able to, you know, dance with her at a prom or something... You can never put a price tag on that.” (Luke Rosen, [45:44])
- “Oh, I think you're gonna get that dance.” (Becky Quick, [45:51])

Key Timestamps

01:33 — Becky Quick: Introduction, personal context (Kaylee's diagnosis)
04:09-07:18 — Luke Rosen: Susanna’s story, KIF1A diagnosis and family impact
08:17 — First connection with geneticist Dr. Wendy Chung, realities of no treatment (Luke Rosen)
14:21 — Humor and spark: the lighter side of Susanna’s personality
15:47-18:39 — Dr. Stan Crook: Explaining nanorare diseases and founding n-Lorem Foundation
20:27 — Dr. Crook’s “gravitational moment” in pursuing one-patient therapies
22:21 — Antisense technology explained
23:25-25:18 — Luke Rosen: The process of securing ASO therapy for Susanna
31:54-33:45 — Observable improvements after treatment; measuring outcomes in non-traditional ways
35:30-37:37 — Addressing pain, quality of life, and the need to focus on "important lanes"
39:49-40:12 — Celebrating victories: singing the National Anthem
42:38-43:58 — Marital and family pressures
45:44-45:54 — Hope for the future; “the dance”

Tone and Style

The episode balances candor, scientific complexity, and raw emotion. Both guests and host share personal moments with vulnerability and warmth, alternating between humor, sorrow, clinical explanation, and advocacy. Their conversation reflects resilience and a pragmatic optimism, offering hope to listeners walking similar paths.

For Further Engagement

Share Your Story or Learn More: cnbc.curesmbc.com
CNBC Cures Newsletter and The Path: CNBC Cures
Upcoming CNBC Cures Summit: March 3rd, New York

BE PART OF THE CONVERSATION:
“Please keep sharing [your stories] and let us know your questions too. Keep coming back.” (Becky Quick, [46:00])

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Transcript

Becky Quick (0:00)

Thy ticket, Lady Jennifer of Coolidge. Well, many thanks, good sir. Here is my Discover card. They accept Discover at Renaissance Fairs? Yeah, they do here. Discover is accepted at the places I love to shop. Get it with the times. With the times. You're playing the loot. Yeah, and it sounds pretty good, right?

Luke Rosen (0:23)

Discover is accepted at 99% of places that take credit cards nationwide. Based on the February 2025 Nielsen report.

Becky Quick (0:31)

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Luke Rosen (1:03)

Well, gosh, if this is the last thing she sees, do I want to be looking at her and smiling or do I want her to know exactly how I feel and that I'm terrified and crying?

Stan Crook (1:12)

Oh, let's see. Can you see?

Luke Rosen (1:17)

There's this purity that isn't the disease, it's the person. This isn't normal and this isn't pain free. Foreign.

Becky Quick (1:33)

I'm Becky Quick. This is the Path from CNBC Cures, a podcast series about the people, the struggles and the science of rare disease. If you listened to or watched our first episode. Thank you. Thank you so much for opening your hearts to my family and our daughter's story. She's one of the millions of Americans with a rare genetic disease. Kaylee's disease is called syngap1. It's a neurodevelopmental disorder that causes conditions like autism and intellectual disability. People with syngap often have difficulty communicating and there is no cure. At least right now. Happy birthday to me.

Stan Crook (2:16)

Happy birthday to me.

Becky Quick (2:20)

Deciding whether to share Kaylee's story was something that my husband Matt and I struggled with for years. Why you don't want to go?

Luke Rosen (2:26)

Well, Kaylee and I get to wrestling.

Becky Quick (2:29)

We are both very private people and we are especially protective of Kaylee. And not being able to ask her consent on this project made us second guess ourselves for a long time. But the outreach and response has been incredible. It's beyond anything either of us has ever experienced. We've received thousands of emails, texts and calls and your comments on YouTube and across social media. Many of you shared your stories about children like Kaylee who or about people you love who are walking this Path of rare disease. Please let's keep this conversation going. We've set up a new email address. It's cnbc.curesmbc.com you can email us to share your story or to learn more about the CNBC Cures Initiative. But the response has been overwhelming and gratifying. I called this series the Path for a few reasons. Rare and the life around it is not one and done like my daughter's therapies that are very targeted. It's repetition, reinvention, recommitment again and again and again. Today's story is about two heroes who have done just that. And they have offered hope, brought by courage, by pioneering science, and by astonishing bravery. Luke Rosen is a hero to me. He is such a good friend. And in another life, he played heroes on television. He also happens to be a real life firefighter in New York. I first met Luke Rosen a couple of weeks into my daughter's syngap1 diagnosis. And he was wonderful. He answered my questions patiently, thoughtfully. He gave me hope.

Luke Rosen (33:45)

Yeah. And we made mistakes with that trial. Just one mistake. And, and that was one of the tools they were using to measure her ambulation is something called the six minute walk test. You walk for six minutes around these cones and. But one thing that this treatment wasn't targeting, wasn't affecting is Susan. The disease does, you know, she was losing her ability to walk slowly. And right now she's fully in a wheelchair. So that measure slowly moved. It doesn't matter to us because, you know, we're not going to force her out of her wheelchair and try to get her to walk. Right. Her fuel tank was on empty every day at 2:00pm yeah. And she would be writhing in pain because she had this. And that's the hardest part is she had. Has this horrible peripheral neuropathy. And so she was writhing in pain. And in her. In her own way, she would wake up and say, you know, my feet are burning on the inside. And was a hard. The pain. Seeing your daughter in pain, I mean, you know, is just. But her peripheral neuropathy started going away, so those painful mornings were gone. If you can eliminate pain, like, God, that was. That's it. You know, I don't have to see her in pain anymore. And she would come home from school at. Barely make it through a couple of hours of school, and then she would just be in bed because she was having seizures all night in her sleep and wasn't getting the right sleep. And so that went away. She was a little bit more. She was more energetic and focused and slowly could make it through an entire day of school. So she wasn't in pain or as much pain. She was making it through school. Breakfast wasn't such a catastrophe. And she was able to sit through a baseball game and sing songs that. And. And we are so fortunate that our daughter, out of all the challenges she has that, you know, she can express herself in ways that I know others can't. And I never take that for granted, because that is one thing that is just, you know, we look at it. And this is something that Dr. Chung had told me too, when I said, how do we. How should we look at this? Like, what is the decision? How should we look at this? And she said, it's like a highway with five lanes on it. And those lanes are, you know, cognition, fine motor, gross motor, all these different, you know, sight. Suzanne is losing her vision right now, which is really hard. And you need to focus on just a few of those lanes because for you, those lanes are going to start falling off the highway. And so we did. We had somebody say, you know, we were able to decide, well, let's focus on communication and let's focus on. Let's focus on maybe cognition or you didn't know what. What to expect. Losing sight is really hard because how do you give somebody. And this is. How do you give a kid the ability to, you know, see what we can see out the window? And then. And then within a year, take it away. You take away. It's not fair.