Rephonic
Artwork for Patient Empowerment Program: A Rare Disease Podcast
N-Lorem Foundation
N-Lorem
Antisense Oligonucleotides
Antisense Technology
NANORARE Mutations
Nano-Rare Diseases
Rare Diseases
Rare Genetic Diseases
Health
Ion Channels
Genetic Testing
Epilepsy
Patient Empowerment
FUS ALS
Nano-Rare Mutations
Hongene Biotech
KIF1A
Motor Neuron Disease
Antisense Oligonucleotide Therapeutics
Neurology

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 na... more

PublishesTwice monthlyEpisodes89Founded4 years ago
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ScienceLife SciencesEducation

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Artwork for Patient Empowerment Program: A Rare Disease Podcast

Latest Episodes

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 ... more

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 applic... more

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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 indust... more

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... more

YouTube

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Recent Guests

Natacha Gassenbach
Senior Vice President of Corporate Affairs and Chief Communication Officer at Biogen
Biogen
Episode: Natacha Gassenbach of Biogen: 2025 Hero of n-Lorem
Susan Hoyt
Director of Publications at Institute for Trial Advocacy
Episode: Seeing with Grace: A Teen’s Journey with a Rare Vision-Affecting Disease
Grace Hoyt
High school sophomore with a rare vision-affecting disease
Episode: Seeing with Grace: A Teen’s Journey with a Rare Vision-Affecting Disease
Brady Huggett
Features editor at The Transmitter, focused on neurosciences and neurodegenerative diseases
Episode: Sneak Peak of the 2025 Nano-rare Patient Colloquium
Luke Rosen
Father of Susanna, a patient with nanorare mutations
Episode: Meet Rescue 7: Firefighters Serving Patients
Frank Vrtochnick
Detective for the Milwaukee Police Department and father of Gunnar, a patient with nanorare mutations
Milwaukee Police Department
Episode: Meet Rescue 7: Firefighters Serving Patients
Raena Vrtochnick
Mother of Gunnar and part of the advocacy work surrounding Rescue 7
Episode: Meet Rescue 7: Firefighters Serving Patients
Kinsley Marta
A four-year-old girl diagnosed with TUBB4A genetic disease
Episode: Hope for Kinsley: Facing TUBB4A with Courage and Care
Hadley Marta
Sister of Kinsley
Episode: Hope for Kinsley: Facing TUBB4A with Courage and Care

Host

Stanley T. Crooke
Host and Founder, CEO, and Chairman of the n-Lorem Foundation. A leading figure in the development of antisense technology and focused on providing innovative, personalized experimental treatments for patients with nano-rare diseases.

Reviews

5.0 out of 5 stars from 50 ratings
  • So informative and important!

    What the n-Lorem foundation is doing for rare disease is a game changer. Hearing from Stan Crooke and the experts and families he interviews is so informative.

    Apple Podcasts
    5
    Flygirl3663
    United States3 years ago
  • Nano what?

    Prior to n-Lorem, patients who didn’t fit into the current healthcare model, were isolated and alone. With this podcast, these patient are front and center. A podcast for and about them! And a master class in basic drug chemistry that anyone would benefit from.

    Apple Podcasts
    5
    Friendly?
    United States4 years ago
  • Excellent resource for patients. Very clear!

    Thanks to Dr. Stanley Crooke and nLorem Foundation for an excellence resource for people living with diseases, their families and for all who want to learn more about BioPharma.

    Best wishes!

    Apple Podcasts
    5
    lalitafer
    United States4 years ago
  • Fantastic

    Very exciting news

    Apple Podcasts
    5
    papanormie
    United States4 years ago
  • Outstanding

    Liked learning more about the rare diseases.

    Apple Podcasts
    5
    ealinek
    United States4 years ago

Listeners Say

Key themes from listener reviews, highlighting what works and what could be improved about the show.

There is a strong emphasis on the importance of community support and advocacy, with many expressing gratitude for the resources provided through the foundation.
Reviewers highlight the uniqueness of the podcast's focus on nano-rare diseases and the urgency of addressing rare genetic conditions, making it a crucial educational resource.
Listeners appreciate the informative nature of the episodes, noting how the discussions bridge the gap between complex medical concepts and personal stories of patients and families.
Many comments reflect on the emotional depth and inspiration drawn from personal experiences shared in the episodes, resonating with both patients and caregivers.

Chart Rankings

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Talking Points

Recent interactions between the hosts and their guests.

Connor Gooley's Story: A First for TUBB4A Treatment
Q: What kind of progress have you seen since starting treatment?
After four doses, they are noticing improvements in Connor's movements, like stability in his head and some increase in control, making them cautiously optimistic.
Connor Gooley's Story: A First for TUBB4A Treatment
Q: How has Connor's condition affected your life?
Initially, it was tough, especially during COVID. The uncertainty fluctuated between hope and despair, but eventually, they found a community and support that made things manageable.
Hope for Kinsley: Facing TUBB4A with Courage and Care
Q: When did you actually begin to fear that she might have something, a problem?
Sarah and Ryan recounted an incident when they visited an ophthalmologist who suggested checking Kinsley's vision, leading to a diagnosis of a rare genetic condition.
Your Questions, Answered: 2026 Q&A
Q: Are there plans for n-Lorem to partner in developing a licensed drug?
Currently, there is no commercial pathway for nanorare ASOs, but there is a movement to establish one.
Your Questions, Answered: 2026 Q&A
Q: What is the frequency range of intrathecal and or LP administration?
The ASOs are administered intrathecally on a defined schedule, typically starting monthly and then moving to quarterly.

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Frequently Asked Questions About This Podcast

What is This Podcast about and what kind of topics does it cover?

This podcast is dedicated to the community of individuals affected by nano-rare diseases, focusing on innovative treatments and insights from leading experts in the field. Each episode features interviews with physicians, scientists, and patient advocates, providing listeners with a deeper understanding of the unique challenges faced by patients with conditions that affect fewer than 30 individuals globally. The discussions often center around the groundbreaking work being done with antisense oligonucleotide technology, highlighting how this approach can lead to personalized, experimental treatments for these rare genetic disorders.

What sets this podcast apart is its commitment to engaging storytelling, where personal accounts of patients... more

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1. This American Life

How many episodes of this podcast are there?

this podcast launched 4 years ago and published 89 episodes to date. You can find more information about this podcast including rankings, audience demographics and engagement in our podcast database.

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What guests have appeared on this podcast?

Recent guests on this podcast include:

1. Natacha Gassenbach
2. Susan Hoyt
3. Grace Hoyt
4. Brady Huggett
5. Luke Rosen
6. Frank Vrtochnick
7. Raena Vrtochnick
8. Kinsley Marta

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