Patient Empowerment Program: A Rare Disease Podcast

n-Lorem Foundation (Dr. Stan Crooke, Amy Williford, Kim Butler, Andrew Serrano, Jon Magnuson, and Kira Dineen)
  • 5.0 (35)
  • Life Sciences

Join the nano-rare disease community! Interviews features leading physicians, scientists, biotech experts, and patient advocates. Lessons teach core concepts about drugs. Our host Dr. Crooke has led the creation of antisense technology and his foundation, n-Lorem, is using this powerful technology to discover, develop, and provide personalized experimental antisense oligonucleotide medicines to nano-rare patients for free, for life. n-Lorem is a non-profit organization established to apply the efficiency, versatility and specificity of antisense technology to charitably provide experimental antisense oligonucleotide (ASO) medicines to treat patients (less than 30 patients) that are the result of a single genetic defect unique to only one or very few individuals. The advantage of experimental ASO medicines is that they can be developed rapidly, inexpensively and are highly specific. n-Lorem was founded by Dr. Stan Crooke, who founded IONIS Pharmaceuticals in 1989 and, through his vision and leadership, established the company as the leader in RNA-targeted therapeutics. The podcast is produced by n-Lorem Foundation and hosted by Dr. Stanley T. Crroke, who is the Founder, CEO and Chairman. Our videographer is Jon Magnuson. Our producers are Kira Dineen, Jon Magnuson, Kim Butler, and Amy Williford. To learn more about n-Lorem, visit nlorem.org. Contact us at podcast@nlorem.org.

  1. 22h ago

    Why n-Lorem Must Sometimes Say No

    We're addressing perhaps the most difficult decision n-Lorem has to make in our work, declining a patient application. Today, our acceptance rate is ~59%. These decisions are made with heavy hearts at n-Lorem, and we are aware of how heartbreaking the decision to decline an application may be for a patient and their family. In this episode, we'll discuss the main reasons a patient's application may be denied, including safety, geography, and other factors.  On This Episode We Discuss: 1:43 – Non-technical issues: Outside of USA, inability to find treating physician, patient population size, if the patient is being treated by another experimental medicine, including repurposed drugs  7:15 – Definitions to better understand the conversation: Heterozygous mutation, haploinsufficiency  9:40 – n-Lorem must understand the cause of the disease, the nature of the mutation, and n-Lorem must be confident that the ASO designed would deliver value to the patient  12:35 – n-Lorem must be confident that only a single gene is causing phenotypes, and that the target is not a null mutation  13:37 – n-Lorem may accept patients, but then discontinue a program if the patient cells are unable to be grown for lab use and testing  14:56 – For patients requiring an allele-selective ASO, n-Lorem must identify non-pathogenic mutations to attempt to create an ASO capable of helping the patient    Links: Donate - https://www.nlorem.org/donate/    Sponsors: Hongene - https://www.hongene.com/  GondolaBio - https://gondolabio.com/

  2. Sep 10

    Sneak Peek of the 2026 Nano-rare Patient Colloquium

    A lot can happen in a year, and at n-Lorem, this past year has brought important milestones, new developments, and plenty to discuss at the upcoming 2026 Nano-rare Patient Colloquium.  In this special Colloquium preview episode, Brady Huggett, editor-in-chief of Asimov Press and longtime moderator of the Colloquium’s patient experience panel, returns to sit down with n-Lorem CEO Stan Crooke. Together, they reflect on the past year, explore some of the topics likely to take center stage, and preview what attendees can expect at the 2026 Nano-rare Patient Colloquium.  On this episode: 2:28 – What is a day in the life of an n-Lorem research team member?  9:08 – How many ASOs are required to move into tolerability studies and what may cause a program to be terminated  12:05 – Improvements in efficiencies, bringing down costs, and growth have allowed n-Lorem to respond to the extraordinary demand  13:14 – Discussing the creation of individualized ASOs for two boys with SCN2A mutations and their potential to help others with the same mutation and single nucleotide variant  25:00 – Thoughts on the FDA’s Plausible Mechanism Framework  29:00 – Commercial opportunities will not alter the n-Lorem charitable arm but will provide sustainable revenues to charitably treat more patients  36:05 – What are the Limits of Hope and expanding those limits  37:54 – Empathy is an expanded sense of self, and a sphere of oneness is felt at the Colloquium    Links: 2026 Nano-rare Patient Colloquium  Donate to n-Lorem   Episode and NRPC Gold Sponsors: ChemGenes GondolaBio Hongene Biotech

  3. Aug 26

    Realities of the Nano-rare: Siblings, Unfairness and Hard Truths with Sally Jackson

    Sally Jackson is a former actress, cookbook co-author, and mother of Susannah. In this Realities of the Nano-rare episode, Sally lets us into her family’s bubble, speaking candidly about the deeply complex and often terrifying realities of navigating KIF1A-associated neurological disorder. From helping Susannah's sibling understand the harsh consequences of her disease to confronting distress, uncertainty, and the profound unfairness of it all, Sally shares the difficult truths her family has faced and continues to carry and fight through. On this episode: 4:00 – Sally and her husband's story first began in a college acting class 9:55 – Forming a career alongside celebrity chef Bobby Flay 13:30 – Family of four including Nat and Susannah 16:55 – Early abnormalities and a diagnosis initially withheld from the family 22:50 – The fight for Susannah and others with KIF1A post-diagnosis 29:24 – Dealing with the seriousness of a degenerative condition while trying to live a normal life 34:00 – Enduring relentless seizures 38:31 – The reality that not even doctors know what's best and often experiment to attempt to relieve rare disease symptoms, and the unfairness of rare disease 45:00 – Informing siblings about the severity and consequences of rare disease 57:00 – Changing the course of their life to advocate to the fullest extent 1:02:25 – After the long fight, there was an ASO 1:07:30 – After years of treatment, Susannah had to stop, a crushing blow that was followed by worsening symptoms before once again resuming treatments Links: Nano-rare Patient Colloquium 2026 Support n-Lorem with a donation Today's Sponsor - Hongene

    Realities of the Nano-rare: Siblings, Unfairness and Hard Truths with Sally Jackson

  4. Aug 5

    Silencing ALS with Neil Shneider

    Silence ALS is an initiative that brings together Columbia University and n-Lorem to support the discovery, development, and treatment of individuals living with nano-rare genetic forms of amyotrophic lateral sclerosis (ALS) through personalized antisense oligonucleotide (ASO) medicines. Learn more about this initiative and one of its co-founders, leading ALS physician-scientist Dr. Neil Shneider in this episode of the Patient Empowerment Program podcast. On this episode we discuss: 4:00 – Developing tools to discuss with patients about the real and scary outcomes of ALS, and changing the course of a neurogenerative and fatal disease 6:52 – Experimental ASO treatments for genetic forms of ALS 9:30 – The creation of Silence ALS to treat extremely rare forms of genetically caused ALS like CHCHD10 and TARDBP and moving the collaboration forward 12:36 – Serving present day and future patient populations with the Silence ALS initiative 17:30 – Learnings gained from treated nano-rare ALS patients may translate to broader ALS groups 22:33 – To ALS patients and families, hope is powerful and makes a difference   Links:  Donate - https://www.nlorem.org/donate/  NRCP26 - https://www.nlorem.org/nano-rare-patient-colloquium-2026/  Hongene - https://www.hongene.com/

  5. Jul 15

    Miracles of Science: Antisense Technology

    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/

  6. Jul 1

    The Personal Rare Disease Journey Behind CNBC Cures with Becky Quick

    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 ----- 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/

  7. Jun 17

    Patient Story: Understanding ALS with Bill O'Sullivan and Neil Shneider, M.D., Ph.D.

    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

  8. Jun 3

    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.

See All (105)

Trailer

5
out of 5
35 Ratings

About

Join the nano-rare disease community! Interviews features leading physicians, scientists, biotech experts, and patient advocates. Lessons teach core concepts about drugs. Our host Dr. Crooke has led the creation of antisense technology and his foundation, n-Lorem, is using this powerful technology to discover, develop, and provide personalized experimental antisense oligonucleotide medicines to nano-rare patients for free, for life. n-Lorem is a non-profit organization established to apply the efficiency, versatility and specificity of antisense technology to charitably provide experimental antisense oligonucleotide (ASO) medicines to treat patients (less than 30 patients) that are the result of a single genetic defect unique to only one or very few individuals. The advantage of experimental ASO medicines is that they can be developed rapidly, inexpensively and are highly specific. n-Lorem was founded by Dr. Stan Crooke, who founded IONIS Pharmaceuticals in 1989 and, through his vision and leadership, established the company as the leader in RNA-targeted therapeutics. The podcast is produced by n-Lorem Foundation and hosted by Dr. Stanley T. Crroke, who is the Founder, CEO and Chairman. Our videographer is Jon Magnuson. Our producers are Kira Dineen, Jon Magnuson, Kim Butler, and Amy Williford. To learn more about n-Lorem, visit nlorem.org. Contact us at podcast@nlorem.org.

Information

  • Creator
    n-Lorem Foundation (Dr. Stan Crooke, Amy Williford, Kim Butler, Andrew Serrano, Jon Magnuson, and Kira Dineen)
  • Years Active
    2022 - 2026
  • Episodes
    105
  • Rating
    Clean
  • Copyright
    © Copyright 2024 n-Lorem Foundation
  • Show Website
    Patient Empowerment Program: A Rare Disease Podcast