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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

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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38:55

Connor Dalby's Story: n-Lorem Patient #001 with...

Patient Empowerment Program: A Rare Disease Podcast ·

2025/04/16

Connor Dalby was born with an SCN2A ion channel mutation causing severe epilepsy and eventually leading to hospice care at an early age.It was a chance hallway conversation between Connor’s mom and Dr. Stan Crooke at a conference that sparked the idea for what would become n-Lorem. In a full-circle moment, Connor went on to become the very first patient applied to and accepted by the foundation, giving him the designation 'Patient #001'. In March 2024, Connor received his personalized ASO

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41:36

Our Mission is Personal with Sarah Glass

Patient Empowerment Program: A Rare Disease Podcast ·

2025/04/02

For n-Lorem's Chief Operating Officer, Sarah Glass, the mission of n-Lorem is deeply personal. Her son Ethan was diagnosed with a nano-rare mutation, a journey that has shaped her commitment to the cause. A geneticist by training, Sarah joined n-Lorem to help lead and guide the organization in its efforts to offer hope and potential help through treatment opportunities to nano-rare patients and their families. This is more than just a job for her—she is driven by her love for her son and a passion

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21:11

How Cells Phone a Friend: Local Communication

Patient Empowerment Program: A Rare Disease Podcast ·

2025/03/19

Our bodies are like bustling cities of cells, always chatting and working together. They don’t just send long-distance messages to organs—they also gossip locally, getting nearby cells to spring into action. Juxtacrine communication is like a handshake between cells—they have to be up close and personal to pass the message along! Unlike long-distance cellular calls, juxtacrine signaling requires direct contact, where one cell’s surface proteins interact with another’s, triggering a response.This

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36:12

How Cells Phone a Friend: Long Distance Communication

Patient Empowerment Program: A Rare Disease Podcast ·

2025/03/05

How do cells communicate with each other? With trillions of cells in the human body, seamless collaboration is essential within this intricate cellular society. Cells work together to form organs, and when one organ needs another to perform a task, they send signals across distances—much like picking up the phone and calling someone. Organ Hotline 📞 Eyes: Hey, Brain? This is Eyes. We’re reading this post about how organs communicate, and it’s really interesting. 📞 Brain: Oh, show me more

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49:30

Rosie’s Story: A Nano-rare Treatment Pioneer

Patient Empowerment Program: A Rare Disease Podcast ·

2025/02/19

Rosie was the first of three HNRNPH2 patients to receive treatment with an n-Lorem ASO, helping to pave the way for others. Her parents, Nicole and Bobby, share their daughter's journey and how they have adapted as a family to meet her unique needs. As a pediatrician, Nicole understands the complexities of medical care, yet she acknowledges that even she doesn’t always have the answers when it comes to raising a child with a nano-rare disease—a reality that resonates with many nano-rare

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53:31

Sloane's Story: A Shared Medicine

Patient Empowerment Program: A Rare Disease Podcast ·

2025/02/05

Sloane was born with a KIF1A mutation and is a special case at n-Lorem—she was the first patient to receive a medicine originally designed for someone else. Her mother, Megan, and grandfather, Tom, join the Patient Empowerment Program to share her journey and provide an update on how she’s doing six months after treatment. On This Episode We Discuss: Sloane’s KIF1A diagnosis Nano-rare diseases affect the entire family Sloane was able to use the same ASO as another KIF1A patient Finding n-Lorem

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45:30

5-years of n-Lorem: What Have We Learned?

Patient Empowerment Program: A Rare Disease Podcast ·

2025/01/22

Five years of creating medicines, instilling hope, and overcoming steep challenges—all with the singular goal of improving the lives of nano-rare disease patients. Along the way, we’ve proven that WE CAN treat these patients safely and effectively, delivering significant benefit. But what else have we discovered throughout this journey? Thank you to Hongene Biotech Corporation for sponsoring this 5-Year Anniversary episode. On This Episode We Discuss: - We CAN do this - n-Lorem is proof of the

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33:27

I Sing the Body Electric

Patient Empowerment Program: A Rare Disease Podcast ·

2025/01/08

Walt Whitman’s 1855 poem, I Sing the Body Electric, is a celebration of the human body and its intrinsic connection to the universe. During that era, scientists were just beginning to understand that humans are, indeed, electrical beings. Whitman’s deliberate use of the word "electric" in his tribute to the human body was quite remarkable, as the term was not widely used at the time and electricity for human use was still in the experimental stage. With that in mind, the poem’s title serves as both

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