Patient Empowerment Program: A Rare Disease Podcast

Miracles of Science #3: RNA World Part 3

In this episode of the n-Lorem Patient Empowerment Program Podcast, host Stan Crooke explores the fascinating world of RNA biology and explains how different types of RNA work together inside cells. He breaks down the roles of ribosomal RNA, messenger RNA, transfer RNA, and several regulatory and processing RNAs, showing how they help convert genetic information into proteins and control cellular function. The episode also highlights how advances in RNA science are helping the n-Lorem Foundation develop individualized treatments for nano-rare patients and their families. 
0:00 – Introduction to the mission of n-Lorem and hope for nano-rare patients and families.  
0:51 – Stan Crooke introduces the podcast and explains n-Lorem’s groundbreaking treatment model.  
1:12 – Overview of the RNA world and why different RNA types are essential to biology.  
1:48 – Introduction to translational RNAs and their role in making proteins.  
2:16 – Explanation of ribosomes and how they translate genetic code into proteins.  
5:12 – Discussion of pre-ribosomal RNA processing and the role of the nucleolus.  
7:48 – Ribosomes are compared to the Enigma machine for decoding biological information.  
9:29 – Overview of transfer RNAs (tRNAs), codons, and amino acid delivery.  
12:23 – Why decoding the genetic code was a landmark scientific breakthrough.  
14:18 – How tRNAs mature through RNA processing and splicing mechanisms.  
16:03 – Summary of how mRNA, rRNA, and tRNA work together during translation.  
18:37 – Introduction to processing RNAs and spliceosome machinery.  
21:15 – Explanation of U4 RNA and its role in RNA quality control and disease.  
23:00 – Overview of snoRNAs and their role in ribosomal RNA maturation.  
24:06 – Discussion of Cajal body RNAs and RNA modification systems.  
25:01 – Introduction to regulatory non-coding RNAs and antisense RNAs.  
27:50 – Overview of circular RNAs and their potential regulatory functions.  
28:51 – Explanation of microRNAs and how they regulate protein production.  
29:59 – Closing thoughts on regulatory RNAs and cellular control systems. 

Miracles of Science #3: RNA World Part 2

In Part 2 of the n-Lorem Patient Empowerment Program podcast Miracles of Science series on RNA, Dr. Stan Crooke further explores the fascinating “RNA world,” explaining how RNA molecules are processed, protected, modified, and used to manage cellular function. The episode breaks down concepts like pre-RNAs, poly-A tails, 5’ caps, RNA splicing, and the multiple “codes” that make RNA dynamic and information-rich — foundational science that helps power antisense therapies for nano-rare patients and helps make the RNA world a true "Miracle of Science," allowing n-Lorem to fulfil its mission. 
 
Shownotes:  
0:00 — Introduction to n-Lorem Overview of the n-Lorem Foundation mission and support for nano-rare patients and families. 
0:15 — Podcast & Sponsor Introduction Dr. Stan Crooke introduces the podcast and acknowledges sponsor ChemGenes. 
1:17 — Entering the “RNA World” Dr. Crooke introduces RNA biology and explains why RNA science is foundational to antisense therapies. 
1:31 — What Are Pre-RNAs? Explanation of immature “pre-RNAs” and how they contain instructions for cellular processing and transport. 
3:31 — RNA Processing & Adding Nucleotides Discussion of how RNAs are trimmed and modified during maturation. 
4:03 — Poly-A Tails Explained What poly-A tails are, why they are added to messenger RNA, and how they protect RNA molecules. 
7:07 — Protecting RNA from Degradation How cells chemically protect both ends of RNA molecules. 
9:00 — The 5’ Cap Introduction to RNA “caps” and their role in identifying and stabilizing messenger RNA. 
11:02 — RNA Splicing How cells remove unnecessary RNA segments (introns) and reconnect useful coding regions. 
14:06 — RNA Structural Codes How RNA folds into structures and why shape is important for function. 
16:09 — Chemical Modification Codes Overview of RNA chemical modifications and how they add another layer of cellular regulation. 
18:22 — RNA as Cellular Management Dr. Crooke compares RNA molecules to executives and middle managers directing cellular operations. 
19:12 — RNA & Antisense Technology Why understanding RNA biology is critical for developing antisense therapies for nano-rare diseases. 
22:08 — DNA vs. RNA Analogy DNA as the “king” and RNA as the active workforce managing the affairs of the cell. 
24:10 — The Dynamic Nature of RNA How RNA constantly changes structure, interactions, and function over time. 
26:14 — Closing Thoughts Final overview of RNA versatility and its role in cellular communication and regulation. 
26:54 — About n-Lorem Information about n-Lorem’s mission to provide personalized experimental treatments free for life to nano-rare patients. 

Miracles of Science #3: RNA World Part 1

In this episode of the n-Lorem Patient Empowerment Podcast, Stan Crooke explores the “RNA World” — the groundbreaking field of RNA biology that helped make modern genetic medicine possible. Drawing from personal experience, he shares the scientific battles surrounding early RNA discoveries, the development of antisense technology, and how decades of persistence ultimately led to life-changing therapies for nano-rare patients. Along the way, Stan explains how evolution, molecular biology, and information systems inside cells shape human health and the future of medicine. 
 
00:00 – Introduction & Sponsor Message Stan introduces the episode and explains the importance of the RNA World to nano-rare medicine. 
02:15 – Why RNA Science Changed Everything How RNA discoveries became foundational to modern genetic therapies. 
05:10 – The Scientific Wars Over Small Nuclear RNAs The fierce debates among scientists about whether snRNAs were real or experimental artifacts. 
09:40 – What Science Is Really Like Behind the Scenes Stan discusses the emotional and competitive nature of scientific discovery. 
13:00 – Antisense Technology & the Road to Spinraza How decades of RNA research led to breakthrough treatments for spinal muscular atrophy. 
17:30 – Lessons From Evolution Why evolution reuses successful molecular strategies to create complex life. 
22:15 – Families of Genes, Proteins & Molecular Efficiency How biological systems adapt and customize molecular functions. 
26:00 – Life as an Information System Stan explains DNA, RNA, proteins, and cells through the lens of information theory. 
29:00 – Final Thoughts: Humanity Wins Through Science Why scientific perseverance ultimately benefits patients and society. 

Realizing Hope for Layken

A nano rare diagnosis reshapes an entire family. 
In Realizing Hope for Layken, Stan Crooke speaks with Callan Pleasant about her daughter Layken and their journey with HNRNPH2. 
Callan shares the early signs, the long road to diagnosis, and what it takes to navigate care while holding onto hope. Through n-Lorem, Layken’s story is moving forward with new possibility. 
Listen to the full conversation with Callan Pleasant. 
Show Notes: 
1:20 – Introduction of special guest Callan Pleasant and her family, namely her daughter Layken 
3:50– Parenthood and speed bumps 
7:30 – First medical consultation regarding Layken 
9:30 – No such thing as a lazy baby. A second look at Layken 
10:40 – Finding a diagnosis: HNRNPH2 
13:50 – Feeling alone after realizing Layken has a nano-rare disease 
16:30 – Managing frustrations and blaming themselves 
20:30 – Managing expectations 
23:00 – Challenging Layken to communicate and move 
26:30 – Finding solidarity with other families 
29:30 – London, Layken's sister 
38:40 – An explanation of HNRNPH2 along with signs and symptoms 
47:30 – Considering possible treatments and risks 
49:30 – Taking a leap of faith and submitting for ASO treatment 
54:00 – n-Lorem providing hope through science 
56:00 – Layken's first treatment 
57:30 – Layken's progress 
58:50 – Positive verbal progression 
1:01:50 – Callan's advice to families 

Realities of the Nano-rare: Episode 2 Oliver Glass

A nano-rare diagnosis changes more than one life—it transforms an entire family. 
In this episode of Realities of the Nano-rare, n-Lorem CEO Stan Crooke sits down with Oliver Glass, Ph.D., MHSc, for an honest and heartfelt conversation about raising a child with DYRK1A syndrome. Together, they discuss the early signs something was wrong, the long search for answers, the realities of daily life, and how their family has adapted to build a new normal. 
From daily challenges to unexpected lessons in resilience, love, and perspective, this episode offers a powerful look inside one family’s journey with rare disease. 
🎧 Listen now and subscribe for more conversations from the nano-rare community. 
Learn more about n-Lorem: https://www.nlorem.org 
Episode Chapters 
0:00 Intro 
1:30 Stan introduces Oliver Glass and his family’s story 
7:30 Early signs and symptoms 
14:20 Running tests and searching for answers 
16:25 The diagnosis journey 
23:00 What is DYRK1A? 
27:45 Life with Ethan today 
32:40 How life looks different as a family 
34:00 When the Glass family first heard about n-Lorem 
36:20 Biggest worries for the future 
38:45 Supporting siblings and balancing attention 
43:00 Advice, reflection, and hindsight 
#RareDisease #DYRK1A #GeneticDisorders #PatientStory #Podcast #nLorem #CaregiverJourney #FamilyStory 

Realities of the Nano-rare: Episode 1 with Sarah Glass

A nano rare diagnosis does not just affect one person. It reshapes an entire family. 
Realities of the Nano-rare takes you inside that experience. Hear directly from n-Lorem COO and nano rare mom Sarah Glass as she joins host Stan Crooke to share the day-to-day realities of raising her son Ethan and the ripple effects on their whole family, including his older sister. 
Discover how they have adapted, what they have learned, and how they have built a new normal. 
Tune in to this powerful two-part series featuring both Sarah and Oliver Glass. 
 
On This Episode We Discuss: 
5:08 – Introduction to Sarah Glass and her family's Nano-rare experience 
9:00 – Initial challenges of accurately identifying Sarah's son Ethan had a Nano-rare disease 
11:30 – Symptoms and telltale signs that there was something else going on with Ethan 
14:45 – Identifying that Ethan has DYRK1A 
17:20 – Challenges of finding solutions post-diagnosis 
20:20 – Navigating Sarah's son's DYRK1A with her unaffected daughter 
23: 04 – What's a good day with DYRK1A? 
27:01 – What's a bad day with DYRK1A? 
34:30 – Managing frustrations of taking care of someone with a Nano-rare disease 
37:05 – Compromises in professional life 
41:00 – Planning for contingencies  
43:10 – Unexpected tender moments of raising someone with DYRK1A 
47:30 – Advice for managing expectations, future plans, and working with others 
 
Make sure to check out our sponsor Chemgenes!

Miracles of Science #2: Induced Pluripotent Stem Cells

Welcome to part two of our series that pulls back the curtain on the scientific and medical breakthroughs that make n-Lorem possible. We call them miracles and without them, n-Lorem would not exist today.
Every day, our patients benefit from what can only be described as miracles of science. Not the fairytale kind. Not wishes granted overnight. These miracles are earned by humans. Built over centuries by scientists whose discoveries stacked, evolved, and refined over time.
They are the result of human curiosity, persistence, and an ever-deepening understanding of biology, and they’ve fundamentally changed how we see the world and treat disease.
Today’s miracle: Induced Pluripotent Stem Cells or iPSCs
iPSCs are a truly transformative scientific breakthrough that are changing what’s possible for nano-rare patients! They allow us to study, test, and personalize treatments in ways that were unimaginable not long ago. For example, they enable n-Lorem to take easily obtained cells, such as skin cells, grow them and then reprogram them into entirely different cell types of interest, like neurons, cells that exist within the brain and would otherwise be inaccessible without invasive procedures. Incredible!
 
On This Episode We Discuss:
0:53 – What are induced pluripotent stem cells (iPSCs) why they are essential to n-Lorem and personalized medicine 
4:25 – The origins of iPSCs began with curiosity and the discovery and study of cells 
10:19 – Cell differentiation: How a single cell (fertilized egg) can create such a diverse universe of other types of cells 
18:45 – Terminal differentiation: The final stage of a cell's development reaching its final form 
21:45 – The iPSC breakthrough and the doors they’ve opened for treatments 
 
 

Where Emerging FDA Draft Guidance may Lead for Nano-rare Patients

On This episode We Discuss:
2:30 – Andrew realized that finance plays a very large role in drug development and is an obstacle in for rare disease patients getting the treatments they need
4:30 – The triple whammy of drug development
6:22 – Economic decisions when lives are at stake; Balancing the ethical considerations of medicine and finance
12:17 – Small biotech companies are better for innovation, and a distributed investment process is more productive; Innovation comes from orthogonal thinking
22:40 – Very small prevalence disease drug programs tend to get terminated at commercial organizations; Movement toward finding a way to create a commercial path for extremely rare disease drugs
25:45 – FDA draft guidance for potential commercialization of nano-rare medicines; Where the guidance may lead; Value of a statistical life
30:32 – The cost of a nano-rare patient’s care; Courageous moves of the FDA
35:00 – Regulatory processes merging with the advances of science while still protecting patients
 
Andrew W. Lo is the Charles E. and Susan T. Harris Professor at the MIT Sloan School of Management, director of MIT’s Laboratory for Financial Engineering, and principal investigator at MIT’s Computer Science and Artificial Intelligence Laboratory. His healthcare-related research interests include: new financial engineering tools and business models for drug and device development and healthcare delivery, especially for rare and ultra-rare diseases; statistical methods for incorporating patient preferences into the drug approval process; predicting clinical trial outcomes via machine learning techniques; and novel reimbursement models for creating a robust gene and cell therapy ecosystem. He is a co-founder of BridgeBio Pharma, QLS Advisors, Quantile Health, and Uncommon Cures; a director of AbCellera, Atomwise, BridgeBio, Uncommon Cures, and Vesalius; and a member of the advisory board to the American Cancer Society’s BrightEdge Impact Fund. Lo received his B.A. in economics from Yale University and his A.M. and Ph.D. in economics from Harvard University.
 
Patient Empowerment Program host: Stan Crooke, n-Lorem Founder and CEO

Reflections from the n-Lorem Chairman | 2026

In this video message, Chairman Stan Crooke reflects on the close of 2025, six years after founding n-Lorem. He shares his gratitude for the community that made it the organization’s most successful year to date and offers perspective on how progress across every area of the foundation is carrying n-Lorem forward into 2026.
Read the 2026 Chairman Letter

Miracles of Science: Genomics

Today, we’re launching a NEW SERIES that pulls back the curtain on the scientific and medical breakthroughs that make n-Lorem possible. We call them miracles and without them, n-Lorem would not exist today.
Every day, our patients benefit from what can only be described as miracles of science. Not the fairytale kind. Not wishes granted overnight. These miracles are earned by humans. Built over centuries by scientists whose discoveries stacked, evolved, and refined over time.
They are the result of human curiosity, persistence, and an ever-deepening understanding of biology, and they’ve fundamentally changed how we see the world and treat disease.
Today’s miracle: Genomics.
Coming next: iPSCs, the RNA World, and Antisense Technology. The breakthroughs that pushed possibility even further.
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The Patient Empowerment Program podcast is hosted by n-Lorem Founder and CEO, Stan Crooke. Dr. Crooke recently joined the advisory board of the CNBC Cures, an initiative bringing together families, doctors, investors and regulators with one goal in mind: helping to raise awareness of, and improve patient outcomes for, the 30 million Americans suffering from rare diseases. You can join the CNBC Cures Newsletter here: https://www.cnbc.com/cnbc-cures-newsletter/
Today's sponsor is ChemGenes: https://www.chemgenes.com/