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PODCAST

Patient Empowerment Program: A Rare Disease Podcast

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

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

science life sciences

All Episodes

51:29

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

Patient Empowerment Program: A Rare Disease Podcast ·

2026/04/08

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

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30:16

Miracles of Science #2: Induced Pluripotent Stem Cells

Patient Empowerment Program: A Rare Disease Podcast ·

2026/03/25

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

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43:20

Where Emerging FDA Draft Guidance may Lead for...

Patient Empowerment Program: A Rare Disease Podcast ·

2026/03/04

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

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18:13

Reflections from the n-Lorem Chairman | 2026

Patient Empowerment Program: A Rare Disease Podcast ·

2026/02/11

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

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33:10

Miracles of Science: Genomics

Patient Empowerment Program: A Rare Disease Podcast ·

2026/02/04

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

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30:38

Your Questions, Answered: 2026 Q&A

Patient Empowerment Program: A Rare Disease Podcast ·

2026/01/14

Your questions, answered. n-Lorem CEO, Stan Crooke, answers questions directly from the nano-rare community in this special Q&A episode. How often is Drug safety monitored? When is an existing ASO made available to others? Not capping accepted applications and more. Do you have additional questions?Email podcast@nlorem.org. On This Episode We Discuss: 5:40 - What is the frequency range of intrathecal and/or LP administrations? Do the treatments not typically cross the blood brain

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46:39

Best of the 2025 Nano-rare Patient Colloquium

Patient Empowerment Program: A Rare Disease Podcast ·

2025/12/17

In this special Best Of episode, we revisit some of the powerful moments from the 2025 Nano-Rare Patient Colloquium—an extraordinary gathering that brought together more than 875 patients, family members, scientists, physicians, advocates, and industry leaders from around the world, both in person and virtually. This episode highlights moving patient stories, thought-provoking scientific conversations, and community-led discussions that reflect the momentum building across the nano-rare space

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24:47

Natacha Gassenbach of Biogen: 2025 Hero of n-Lorem

Patient Empowerment Program: A Rare Disease Podcast ·

2025/12/03

A conversation with Natacha Gassenbach, 2025 Hero of n-Lorem and Biogen leader. She shares Biogen’s decision to become a founding donor of n-Lorem, the impact of the Nano-Rare Patient Colloquium. Natacha also explores “the movement for nano-rare” and a shared vision of tackling difficult challenges to drive meaningful change.Holiday Ornament: https://www.nlorem.org/holiday-ornament-fundraiser/ On this episode: 1:18 – Natacha introduction 3:00 – Biogen’s path to becoming a founding donor of

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48:46

Seeing with Grace: A Teen’s Journey with a Rare...

Patient Empowerment Program: A Rare Disease Podcast ·

2025/10/08

Meet Grace Hoyt — a bright, witty high schooler with a passion for singing, art, and romantasy audiobooks. Grace also happens to live with a rare disease (FLVCR1) that has left her legally blind, unable to feel pain, and unsteady on her feet.On her 13th birthday, Grace received a first-of-its-kind ASO medicine designed by n-Lorem to preserve her remaining vision. Today, she can still read snippets of her beloved “book children” — the physical versions of her favorite audiobooks — and, importantly,

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36:55

Sneak Peak of the 2025 Nano-rare Patient Colloquium

Patient Empowerment Program: A Rare Disease Podcast ·

2025/09/24

As the 2025 Nano-rare Patient Colloquium approaches, we welcome back Transmitter Features Editor Brady Huggett to guest-host this episode of the Patient Empowerment Program podcast. Brady sits down with n-Lorem founder and CEO, Dr. Stan Crooke, to reflect onto reflect on the strides made over the past year—insights that will help shape the discussions at this year’s Colloquium.On This Episode We Discuss: 2:10 How n-Lorem decides to give a medicine specifically designed for one person to another

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46:27

A Conversation with the 2025 Heroes of n-Lorem

Patient Empowerment Program: A Rare Disease Podcast ·

2025/09/10

The ‘Hero of n-Lorem’ Award is a recognition given to an individual volunteer or organization that has made substantial contributions to n-Lorem and our nano-rare patients. This honor recognizes our ‘unsung heroes’, those who have gone the extra mile, taken time away from their schedules and families and leveraged their expertise and contacts to support n-Lorem’s mission to provide hope and potential help to nano-rare patients. On this episode of the Patient Empowerment Program podcast, we welcome

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47:17

Meet Rescue 7: Firefighters Serving Patients

Patient Empowerment Program: A Rare Disease Podcast ·

2025/08/27

Rescue 7 is dedicated to serving the most vulnerable patient communities with care, compassion, and critical support. Inspired by the traditions of Fire Departments, Police Departments, and Military service worldwide, their programs go beyond emergency response. From patient transportation to sibling support initiatives, as well as emergency disaster relief efforts — Rescue 7 is there. Always ready and there when you need them.Rescue 7 is founded by n-Lorem families. They will host the Family Club

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17:11

Hope for Kinsley: Facing TUBB4A with Courage and Care

Patient Empowerment Program: A Rare Disease Podcast ·

2025/08/06

Sarah and Ryan Marta, proud parents of three girls from Central Texas, began noticing that their middle daughter, Kinsley, was slow to reach mobility milestones in her early years. During a visit to a friend’s house, concerns about her vision were raised, prompting a check-up. What began as a single hospital visit quickly turned into many. An MRI revealed abnormal levels of white matter in Kinsley’s brain—leading to the devastating and confusing diagnosis of a nano-rare neurodegenerative disease

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21:30

New Insights into Genetically Caused Developmental...

Patient Empowerment Program: A Rare Disease Podcast ·

2025/07/23

Emerging observations from our work at n-Lorem suggest that not all developmental delays are permanent. In the absence of structural damage—such as microcephaly, congenital deformities of the skull or bones, or organ malformations—there appears to be potential for improvement in movement disorders, cognition, autistic features, and more. These insights point to a level of resilience and plasticity in the central nervous system that may be greater than previously believed, and they are beginning to

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23:48

Research to Advance ASO Technology at n-Lorem

Patient Empowerment Program: A Rare Disease Podcast ·

2025/07/09

At n-Lorem, every investment decision carries real weight by directly affecting the nano-rare patients we serve. We think very deeply about each decision of how to invest the limited and precious dollars that we have. But what about investing in basic research to advance antisense oligonucleotide (ASO) technology for the future? Certainly, most of the money should and is being put towards creating ASOs to help patients in need today. Meanwhile, it is also our belief that we must invest to continue

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31:24

Commitment to Safety First: The DSMB

Patient Empowerment Program: A Rare Disease Podcast ·

2025/06/25

The Data Safety Monitoring Board (DSMB) is composed of distinguished, independent experts in antisense oligonucleotide (ASO) technology, clinical trial design and evaluation, and drug development. Many are also dedicated clinicians who care for patients with rare diseases. This board provides unbiased safety oversight by reviewing accumulated data from all investigator-initiated studies involving n-Lorem’s ASO medicines.n-Lorem's DSMB is chaired by Dr. Eugene Schneider, Executive Vice President and

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46:49

Connor Gooley's Story: A First for TUBB4A Treatment

Patient Empowerment Program: A Rare Disease Podcast ·

2025/06/11

Connor Gooley is the first patient ever treated with an ASO for TUBB4A-Related Leukodystrophy, a condition that severely disrupts his nervous system, slows nerve impulses, and impairs his fine motor skills. As a result, Connor cannot speak, walk, sit up on his own, or chew well. Still, he manages to army crawl, propel himself in his wheelchair, and use a gait trainer. He’s also remarkably resilient—rarely crying or complaining despite the daily challenges he faces.In this episode of the Patient

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20:08

What Are iPSCs—and Why Do They Matter?

Patient Empowerment Program: A Rare Disease Podcast ·

2025/05/28

Induced pluripotent stem cells (iPSCs) are a groundbreaking, and mind-blowing, scientific advancement—one of many that help make it possible for n-Lorem to do what we do. In short, typical skin cells (such as fibroblasts) are taken from an individual and reprogrammed using specific factors to become iPSCs. These iPSCs are then redifferentiated into any desired cell type in the body, such as muscle or liver cells. You can do that? Yes, and we do! The most common cell type that we use at n-Lorem are

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25:23

Biological Systems & Chemical Networks

Patient Empowerment Program: A Rare Disease Podcast ·

2025/05/14

What Are Chemicals? | How Drugs Work in the Body | Understanding HomeostasisWe’re all made of chemicals—but what exactly is a chemical? In this video, we break down the basics: chemicals are forms of matter that exist as solids, liquids, or gases. Inside living organisms, these chemicals create complex networks that keep us alive and balanced—a process known as homeostasis. Drugs are chemicals too! They work by interacting with these biological networks to help restore or adjust how the body

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29:22

Why ASO Technology?

Patient Empowerment Program: A Rare Disease Podcast ·

2025/04/30

What makes antisense oligonucleotides (ASOs) so special?Let’s first understand what an oligonucleotide is. An oligonucleotide is a short strand of synthetic DNA or RNA (a nucleic-acid chain), usually consisting of up to approximately 20 nucleotides long—designed to bind with specific sequences in the body. At n-Lorem, our ASO technology is built on more than 30 years of research, innovation, and investment. It’s uniquely suited for treating nano-rare diseases—ultra-rare genetic conditions that

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