Symptomatic: A Medical Mystery Podcast

Episode: Case #30: Rose & Casey

Host: Lauren Bright Pacheco
Date: December 2, 2025

Overview:

This episode spotlights Casey McPherson, a rock musician turned parent- a relentless father fighting to save his daughter, Rose, from an ultra-rare neurodevelopmental disorder called HNRNPH2-related developmental disorder. Casey narrates his family’s experience with medical uncertainty, the journey to diagnosis, and ultimately, the decision to try to create a treatment himself—helping pioneer new approaches to rare disease drug development. It’s a story of heartbreak, grit, hope, and the drive to change a broken system for families everywhere facing “impossible” odds.

Main Discussion Points & Insights:

1. The Onset of Rose's Mysterious Symptoms

• Early Signs:
  • At around 18 months, Rose’s development regressed. She lost language, motor skills, and began screaming uncontrollably.
  • Casey recalls, "She was screaming uncontrollably, like she was in pain and confused and angry. You could just see on her face, it was very, very clear that something was going very wrong." (00:03)
• Parental Alarm:
  • Medical professionals dismissed early concerns, attributing issues to normal developmental variation.
• Family Perspective:
  • Weston, Rose's older sister, describes Rose’s vibrant personality and the family's close bond.
    • “She loves animals....once you get to know her, she's a little bit, like, spicy and a little bit sassy.” (05:16)
    • “Her favorite drink is definitely Dr. Pepper. She’s stolen my dad’s Dr. Pepper millions of times.” (05:45)

2. Struggle for Diagnosis

• Obstacles in Healthcare:
  • Insurance limitations meant basic screening panels only tested for diseases with existing treatments. Most rare diseases were not screened.
    • Casey: “It tests for diseases that currently have treatments. It doesn't test for diseases that don't.” (07:57)
• Researching as a Family:
  • The McPhersons self-financed a $10,000 whole exome genetic panel (now $250), collecting family DNA to seek answers.
• Devastating Prognosis:
  • The neurologist, with little empathy, tells them Rose has a rare HNRNPH2 mutation:
    • “There's really nothing we can do, Casey, you know, other than make her comfortable. Good luck.” (12:55)
  • At the time, only 33 known cases worldwide.

3. Understanding HNRNPH2-related Developmental Disorder

• Expert Explanation (Rodney Bolling Jr.):
  • Mutation blocks RNA processing, impacting >400 genetic messages:
    • “It’s like putting on a boxing glove and trying to shake somebody’s hand. It’s not going to happen very well.” (13:37)
• The Impact:
  • Most children never live independently; symptoms include seizures, loss of language, and profound disability.
  • Casey reflects on the grief:
    • “There's a grief that you go through when you know that your child is not going to have the same freedoms and hopes and dreams and capabilities that you and I do....” (14:44)

4. Turning Pain into Purpose—Casey’s Drive to Find a Treatment

• Broken System Realization:
  • “She didn’t have a cure...because nobody felt like they could make enough money off of it.” (15:12)
  • 10,000+ rare diseases, less than 5% with treatments; most lack commercial incentives for drug development.
• Learning & Networking:
  • Casey teaches himself genetics, networks with scientists, and connects with rare disease parent-advocates like Julia Vitarello.
    • “I probably talked to two or three hundred CEOs of biotech companies, drug developers, scientists, and developed a network of people that wanted to help.” (20:46)
• Finding Allies:
  • Forms a partnership with Rodney Bolling Jr., drug developer/engineer, who is moved to leave his career to help.
    • “He’s charismatic and on a mission and it is so easy to fall in behind him and help him carry that mission....That lunch turned into dinner and we started to cure Rose that day.” (23:27)
    • “I have six healthy children and I feel that I owe a debt, an unpayable debt to the fact that I have those six healthy kids.” (24:00)

5. Challenges in Developing a Treatment

• Systemic Obstacles:
  • Contract research organizations, academic bureaucracy, and high costs create huge delays and lost resources.
    • "We paid $30,000 up front...they came back and said...we've lost half the cell lines.” (26:33)
    • On picking up and starting their own lab: “Rodney, we just need to start our own lab." (27:14)
• Resourcefulness:
  • Casey forms AlphaRose Therapeutics, leverages fundraising within his fanbase, church, and the rare disease community.
  • Organizes events: Rose Fest, golf tournaments, and galas to fund science.
    • “The foundation's basically funded by fans of my band, people from my church, people that latched onto Rose's story.” (27:49)
• Expanding the Mission:
  • Casey sees the systemic need:
    • “What about the kids in Australia? What about the kids in France? ...Create a system that once we treated Rose, we would have some sustainable way of doing this so parents didn’t have to pay for this crap anymore.” (28:45)

6. Building a Lab and Preclinical Breakthroughs

• New Model:
  • Set up a lab focusing on parent- and scientist-driven rare disease therapeutics; families own intellectual property.
• Developing the Therapy:
  • Rodney details using Rose’s blood to create stem cells and neurons, then testing 55 antisense oligos (ASO):
    • “In seven of our 55 ASOs, we saw significant progress. And that was an amazing moment.” (29:57)
• Clinical Plans:
  • Preclinical safety studies in rats are underway; the goal is an FDA clinical trial to bring the treatment to more patients.
    • “We found a hospital that we can treat her and some other children in, and we're setting up a proper clinical trial to attempt to get it approved by the FDA so we can create, you know, global access for the drug.” (31:47)

7. Personal Transformation and Impact

• Casey’s Evolution:
  • Once told to “stay in your lane,” he’s learned the science, the business, and the advocacy himself.
    • “I would have loved it to be your job. I never wanted to quit my music career, but here we are because someone didn’t do their job ... I did every single one of those things for Rose.” (32:14)
• Concrete Results:
  • Over 22 families helped; 8 new rare disease drugs developed through their programs.
• Rodney on Casey:
  • “The Casey that I met in 2019 had never passed high school biology and now he knows the language, he's running a pharmaceutical company and is using those connections and that charisma to cure not only his daughter, but create a pathway for everyone with rare disease.” (33:18)

8. Current State & Message of Hope

• Rose’s Present:
  • “She is happy. We've got her Seizures under control ... After this podcast, I'll go snuggle with her in bed ... She’ll let me snuggle with her, which is just amazing. Like, it's my favorite part of the day.” (36:12)
• Weston’s Perspective:
  • “If she didn’t have the condition, then we wouldn’t have helped all these kids.” (35:28)
• Rodney’s Final Word:
  • “How easy would it have been for Casey to claim victimhood and crawled under a rock and hold his daughter and cry? And that's not what he did. He has fought, and as we've struggled, he's continued to fight. And he's invented, literally invented new ways to fight. And his fight has already helped others.” (37:04)
• Casey's Closing Reflection:
  • “We live in this incredible time of technology. This is more of a civil rights issue than anything. We have the technology to treat many of these diseases, but we don't have the systems and business models and support to do it at any sort of scale yet. And as we're building that, I think it's important that families take courage, have the courage to hope, because to have hope means you think that maybe something could change. And that's a scary thought when you're trying to accept the reality of what you're in. And I think it's okay to completely love your child and accept the reality you're in and at the same time not stand for it. I think that's okay.” (39:40)

Memorable Quotes & Timestamps

• “She was screaming uncontrollably, like she was in pain and confused and angry. You could just see on her face, it was very, very clear that something was going very wrong.” — Casey McPherson (00:03)
• “It tests for diseases that currently have treatments. It doesn't test for diseases that don't.” — Casey McPherson (07:57)
• “[The doctor said] 'There's really nothing we can do, Casey, you know, other than make her comfortable. Good luck.’” — Casey McPherson (12:55)
• “It's like putting on a boxing glove and trying to shake somebody's hand. It's not gonna happen very well.”— Rodney Bolling Jr. (13:37)
• "I would have loved it to be your job. I never wanted to quit my music career, but here we are because someone didn't do their job...I did every single one of those things for Rose." — Casey McPherson (32:14)
• “How easy would it have been for Casey to claim victimhood and crawled under a rock ... and that's not what he did. He has fought, and as we've struggled, he's continued to fight...and his fight has already helped others.” — Rodney Bolling Jr. (37:04)
• “We live in this incredible time of technology. This is more of a civil rights issue than anything....I think it's important that families take courage, have the courage to hope…” — Casey McPherson (39:40)

Key Timestamps by Topic

• 00:03–04:56 — Onset & Early Symptoms
• 07:57–12:55 — Diagnosis Odyssey & Confirmation
• 13:37–16:19 — Scientific Explanation & Systemic Gap
• 19:26–23:56 — Networking, Finding Allies, Partnership with Rodney
• 25:39–27:29 — Systemic Obstacles & Lab Creation
• 27:49–29:27 — Fundraising, Community Building, Broader Mission
• 29:57–31:47 — Scientific Breakthrough, ASO Therapy, Preclinical Progress
• 32:14–33:41 — Casey’s Transformation, Impact on Rare Disease Community
• 36:12–37:04 — Rose Today, Message of Family and Hope
• 39:40 — Closing Reflection & Call for Courage

Tone & Emotional Takeaways

The episode maintains a compassionate, candid, and resilient tone. It's marked by moments of heartbreak and clarity, uplifted by hope, tireless innovation, and relentless love. The humor and warmth within family exchanges provide grounding amidst the gravity of the medical mystery.

Resources & Links Mentioned

• alpharos.com
• decurarose.org
• tcarrarelabs.org
• Follow Casey McPherson on Instagram

This episode is a moving testament to the power of family, science, and community—an inspiring push for changing the way ultra-rare diseases are confronted, remembered through the eyes, voice, and fight for a child named Rose.