Patient Empowerment Program: A Rare Disease Podcast

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!

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

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

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

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/