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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

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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24:22

Q&A - 2024 Nano-rare Patient Colloquium Recap

Patient Empowerment Program: A Rare Disease Podcast ·

2024/12/18

Join Stan Crooke, n-Lorem founder, CEO, and host of the Patient Empowerment Program Podcast, alongside Amy Williford, Sr. Director of Communications and Donor Relations, for a special episode.In this post-colloquium Q&A, Stan recaps the 2024 Nano-rare Patient Colloquium and the progress n-Lorem has made as the organization nears its 5-year anniversary, shares his candid reflections on the event, and dives into additional thoughts and questions that weren’t covered. Do you have additional

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58:13

Best of the 2024 Nano-rare Patient Colloquium

Patient Empowerment Program: A Rare Disease Podcast ·

2024/12/04

This year’s Colloquium spanned two perfect autumn days in beautiful Cambridge, Massachusetts where hundreds of nano-rare community members from around the world gathered under a single roof to learn, connect, and support one another. We’re pleased to announce that the event welcomed over 750 attendees, both in-person and virtually. It’s each and every one of you who made the event such a success! For this special episode, we gathered the ‘Best’ clips from the event for you to learn from and

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40:38

Susannah's Progress: Two Years of ASO Treatment with...

Patient Empowerment Program: A Rare Disease Podcast ·

2024/10/16

Two years of treatment—two years of strength, courage, and blazing a trail for other nano-rare patients! 🎉 Susannah's story was the first shared on the Patient Empowerment Program podcast. Now, two years later, her father, Luke Rosen, and her physician, Dr. Jennifer Bain, detail Susannah’s remarkable progress since beginning regular treatments—highlighting improvements in her motor skills, cognition, energy, and more!Susannah's Story (May 25, 2022) Donate 2024 Nano-rare Patient Colloquium On

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41:44

Sneak Peek of the 2024 Nano-rare Patient Colloquium

Patient Empowerment Program: A Rare Disease Podcast ·

2024/10/02

Take a glimpse behind the curtain as we offer you a sneak peek of the 2024 Nano-rare Patient Colloquium. We’re thrilled to welcome back longtime biotech journalist and current Features Editor of The Transmitter, Brady Huggett, to the podcast. In an interview with n-Lorem founder and CEO Stan Crooke, they preview the upcoming Colloquium, diving into key topics that will shape the conversation at the event and around nano-rare diseases in the year ahead.On This Episode We Discuss: 2:21 How many

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29:50

The Building Blocks of What's Possible with David...

Patient Empowerment Program: A Rare Disease Podcast ·

2024/09/18

Hongene Biotech is a producer of RNA building blocks – the first step for what’s possible in RNA drug-discovery and development. David Butler, Ph.D., Chief Technology Officer of Hongene, joins the Patient Empowerment Program to discuss why Hongene aims to help make RNA medicines accessible and affordable for patients everywhere, regardless of prevalence.On this episode we discuss: 2:40 Hongene Biotech and what do they do 8:09 The early days of nucleoside building blocks and maturation of

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29:34

Knowledge is Power: Part 2

Patient Empowerment Program: A Rare Disease Podcast ·

2024/09/03

The concluding episode of the Knowledge is Power series. The knowledge we are gaining at n-Lorem has even more value than benefiting nano-rare patients and their families. These insights will enable scientists to discover new therapeutic targets for both common and rare diseases, fundamentally changing the way we approach health and disease.Survey – Patient Empowerment Program Podcast Register for the 2024 Nano-rare Patient Colloquium On This Episode We Discuss: Questions we can answer and

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35:19

Knowledge is Power: Part 1

Patient Empowerment Program: A Rare Disease Podcast ·

2024/08/21

The knowledge we are gaining at n-Lorem has even more value than benefiting nano-rare patients and their families. These insights will enable scientists to discover new therapeutic targets for both common and rare diseases, fundamentally changing the way we approach health and disease.Survey – Patient Empowerment Program PodcastRegister for the 2024 Nano-rare Patient ColloquiumOn This Episode We Discuss:Two most common words in medicine: Health and DiseaseThe Scientific MethodThe Importance of

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33:39

Adopting a Rare Son with Paul Compton

Patient Empowerment Program: A Rare Disease Podcast ·

2024/08/07

Paul Compton and his wife Andreachoseto start a family through adoption. Unknown to anyone, one of their adopted boys, Taeson, was the inheritorof DRPLA, a progressive brain disorder caused by a mutation in the ATN1 gene. Determined to find a treatment and advocate for their son and others with the disease, the Comptons established CureDRPLA. In early 2024, Taeson received his initialtreatment with an ASO discovered and developed by n-Lorem. Paul discusses his family’s journey and shares his

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30:16

Ion Channel Mutations: The Complexities of Treatment

Patient Empowerment Program: A Rare Disease Podcast ·

2024/07/24

Mutations affecting ion channels are the most common cause of 'applications for treatment' submitted to n-Lorem. These channels regulate the passage of essential electrically charged ions, like sodium, potassium, calcium, and chloride, into cells. Alas, ion channels pose a significant challenge in the ASO discovery process, as there is little room for error due to the need for highly allele-selective ASOs to achieve success. Let’s 'dive into the channel' and explore the complexities of treating

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29:18

Why We Dose Intrathecally

Patient Empowerment Program: A Rare Disease Podcast ·

2024/07/10

The affected organs of our patients determine the route of administration for an ASO, as certain routes can better target specific organs and offer the best potency. The central nervous system (CNS) is the most common target we encounter, and for this, we dose intrathecally. Let’s dive into how this is done and the particulars of why this approach is used. The Human Body and BarriersThe Four Tubes – Enteral, Respiratory, Urinary, and the Central Nervous System (CNS) tubeHow The Body Constructs

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27:33

Behind the ASO Design and Discovery with Konstantina...

Patient Empowerment Program: A Rare Disease Podcast ·

2024/06/26

Konstantina Skourti-Stathaki leads the n-Lorem research team behind the design and discovery of ASOs. Nadina, as we at n-Lorem call her, has a passion for helping patients and an excitement for science that is contagious. She joins the Patient Empowerment Program podcast to discuss her day-to-day activities as n-Lorem’s Director of ASO Design and Discovery, the ongoing research her team is conducting, and more!On This Episode We Discuss:Inspired to study biology at the University of CreteLeaving

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36:51

Best of the 2023 Nano-rare Patient Colloquium

Patient Empowerment Program: A Rare Disease Podcast ·

2024/06/12

We’re recapping the 2023 Nano-rare Patient Colloquium in this special “Best of” episode. For the Colloquium, we gathered the nano-rare community under one single roof for a full day of presentations and panels with experts, partners, supporters, and patients. So, we are bringing some of the top moments directly to your speakers. Discover the origins and aspirations of n-Lorem and Columbia University's Silence ALS initiative, visualize the journey of a nano-rare patient with the help of an emotional

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50:51

The Hurdles of Insuring Patients with Nano-rare...

Patient Empowerment Program: A Rare Disease Podcast ·

2024/05/29

What's preventing insurers from covering the specialized care of patients with nano-rare mutations, thereby expanding access to available treatment options? Is it the population size, cost, or perceived risk? Given that the initial discovery of nano-rare mutations is relatively recent, paving the way for this population requires a nuanced approach to overcome various obstacles.Alan Lotvin, M.D., CEO and co-founder of Sequel Med Tech, and former president of CVS Caremark, conceptualizesthese

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43:06

Anna's Story of Hope and Help: FUS-ALS with Sonja...

Patient Empowerment Program: A Rare Disease Podcast ·

2024/05/15

Stan Crooke speaks with Sonja Kampfer and Dr. Neil Shneider, associate professor of motor neuron disorders at Columbia Medical School, about Sonja’s daughter, Anna. Anna was diagnosed with an aggressive, fatal form of ALS at the age of 16. ALS is rare in teenagers, and this form of ALS is the worst of the worst. Sonja tells Anna’s story initial symptom onset, to diagnosis, and to Anna's response to ASO treatment. As a parent, Sonja reveals her full range of emotions during the duration of her

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50:06

Mostyn's Story: Mother's Day Special with Zoe Hummel

Patient Empowerment Program: A Rare Disease Podcast ·

2024/05/01

Motherhood is a journey filled with challenges, but every step is worth it for the love and joy it brings. Zoe Hummel joins the show in this special Mother’s Day episode to discuss her son Mostyn’s emotional story to diagnosis, and the unique challenges and triumphs of being a nano-rare mom.Order your Mother's Day card by May 6, 2024! All proceed support nano-rare patients.On This Episode We Discuss:How Zoe met her Husband, Mostyn’s fatherZoe’s Violin origins and playing with Rod StewartMostyn’s

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48:53

Physicians and Institutions Transform the Lives of...

Patient Empowerment Program: A Rare Disease Podcast ·

2024/04/17

Research physicians and their associated institutions are monumental to the mission of the n-Lorem Foundation and are truly transformational to the lives of the nano-rare patients they treat. Olivia Kim-McManus, M.D., was one of four physician panelists at our first Colloquium who participated in "A Physician's Perspective on n-Lorem and Nano-rare". Hear Olivia's outlook on nano-rare patients and how the support of an institution like UCSD Rady Children’s Hospital makes all the difference.On This

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25:52

Advanced Genetics Part 2

Patient Empowerment Program: A Rare Disease Podcast ·

2024/04/03

Genetics can be intricate, no doubt. With millions of mutations present in every human, one might wonder why not everyone is impacted by pathogenic diseases. The Patient Empowerment Program aims to assist you in grasping the fundamental concepts of various mutations and how they function, paving the way for you to enhance your understanding of genetics. Delve into the diverse array of disease-causing mutations, their characteristics, and explore which mutations could potentially be addressed through

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30:13

Advanced Genetics Part 1

Patient Empowerment Program: A Rare Disease Podcast ·

2024/03/20

Each time one of the cells in your body divides to form a new cell, 3 BILLION genetic letters must be accurately duplicated. That’s a big number and mistakes DO happen in everyone. That’s right, if you’re reading this, you have a genetic mutation.On This Episode We Discuss:Your genetic alphabet – nucleotidesHow to think about DNA ReplicationTypes of mutationsWhat is an SNP and why you should careIndelsThe genetic codeHow genetic information is translated into a protein

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