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])