The final chapter of our Miracles of Science series has arrived! Today’s miracle is an important one: Antisense Technology, perhaps you've heard of it 😉

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.

This series has pulled back the curtain on the scientific and medical breakthroughs that make n-Lorem possible, including Genomic, Stem Cells, the RNA World and Antisense Technology. Their importance is consequential, and without these breakthroughs, n-Lorem would not exist today.

On this episode we discuss:

- The Evolution of the Drug Discovery and Development Industry

- The Rise of Small-Molecule Drug Discovery

- New Platforms That Expanded the Possibilities of Drug Discovery

- Why Antisense?

- How Ionis Created ASO Technology

Links:

Nano-rare Patient Colloquium 2026: https://www.nlorem.org/nano-rare-patient-colloquium-2026/

Support n-Lorem: https://www.nlorem.org/donate/

Episode sponsors:

Hongene: https://www.hongene.com/

Chemgenes: https://www.chemgenes.com/

We’re celebrating our 100th episode with a special guest, CNBC Squawk Box’s own Becky Quick! Becky’s daughter Kaylie lives with SYNGAP1, and their family’s rare disease experience inspired the creation of 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. Get to know Becky and her daughter Kaylie’s story in this episode of the n-lorem Patient Empowerment Program podcast.

Sign up for the CNBC Cures Newsletter: https://www.cnbc.com/cnbc-cures-newsletter/

On this episode we discuss:

1:25 – Celebrating 100 episodes of the n-Lorem Patient Empowerment Program podcast

6:40 – Welcome Becky Quick; CNBC Anchor and mother of a Kaylie

10:35 – Becky’s journalistic origins and path

16:13 – Launching CNBC Cures and the need to help others facing rare diseases

23:00 – Navigating life with the challenges caused by rare disease

29:03 – Kaylie’s SYNGAP1 diagnostic odyssey

42:10 – Tending with the loss of control

43:56 – Non-verbal does not mean lacking understanding

48:30 – SYNGAP1 explained

59:01 – Hope is powerful

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Make hope possible with a donation in support of nano-rare patient programs: https://www.nlorem.org/donate/

This episode is made possible thanks to our sponsors: Learn more about Chemgenes - https://www.chemgenes.com/

In this episode of the n-Lorem Patient Empowerment Program Podcast, Dr. Stan Crooke is joined by n-Lorem patient Bill O’Sullivan and Dr. Neil Shneider to discuss Bill’s experience living with a rare inherited form of ALS caused by a CHCHD10 mutation. Bill shares his path to diagnosis, the impact of genetic testing, and his experience receiving a personalized antisense oligonucleotide (ASO) treatment designed to target the underlying cause of his disease. Together, they discuss how advances in genetics and precision medicine are creating new possibilities for patients and families affected by rare neurodegenerative diseases.

On this episode we discuss:

• 00:00 Introduction
• 00:58 Meet Bill O’Sullivan and Dr. Neil Shneider
• 03:17 Family history and inherited ALS
• 06:37 Diagnosis and discovery of CHCHD10
• 10:02 Early symptoms and diagnostic challenges
• 20:20 Beginning personalized ASO treatment
• 22:33 Treatment results and disease stabilization
• 23:42 Looking ahead: earlier intervention and future generations
• 27:57 Expanding treatment opportunities through genetic research

Links:

This episode is made possible thanks to our sponsor ChemGenes

Donate to n-Lorem and Make Hope Possible

Register to attend the 2026 Nano-rare Patient Colloquium