Patient Empowerment Program: A Rare Disease Podcast
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. Crooke, who is the Founder, CEO and Chairman. Our videographer is Jon Magnuson. Our producers are Kira Dineen, Jon Magnuson, Andrew Serrano, and Amy Williford. To learn more about n-Lorem, visit nlorem.org. Contact us at [email protected].
- Update frequency
- every 13 days
- Average duration
- 33 minutes
- Episodes
- 83
- Years Active
- 2022 - 2025
Susannah's Progress: Two Years of ASO Treatment with Dr. Jennifer Bain and Luke Rosen
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 y…
Sneak Peek of the 2024 Nano-rare Patient Colloquium
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 …
The Building Blocks of What's Possible with David Butler, Ph.D.
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 Pa…
Knowledge is Power: Part 2
Knowledge is Power: Part 1
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 c…
Adopting a Rare Son with Paul Compton
Paul Compton and his wife Andrea chose to start a family through adoption. Unknown to anyone, one of their adopted boys, Taeson, was the inheritor of DRPLA, a progressive brain disorder caused by a m…
Ion Channel Mutations: The Complexities of Treatment
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 sodiu…
Why We Dose Intrathecally
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) …
Behind the ASO Design and Discovery with Konstantina Skourti-Stathaki, Ph.D.
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 scien…
Best of the 2023 Nano-rare Patient Colloquium
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 …
The Hurdles of Insuring Patients with Nano-rare Mutations with Alan Lotvin, M.D.
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 percei…
Anna’s Story of Hope and Help: FUS-ALS with Sonja Kämpfer and Dr. Neil Shneider
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 aggress…
Mostyn’s Story: Mother’s Day Special with Zoe Hummel
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 e…
Physicians and Institutions Transform the Lives of Nano-rare Patients with Olivia Kim-McManus, M.D.
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 …
Advanced Genetics Part 2
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 a…
Advanced Genetics Part 1
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 y…
Sharing Hope Through Storytelling & Connection with Shanna Tolbert
What's one way to spread hope? By sharing your experiences and connecting with others who truly understand the everyday challenges nano-rare patients face. Shanna Tolbert was one of five nano-rare pa…
Our Mission is Personal with Sarah Glass
For n-Lorem’s Chief Operating Officer, Sarah Glass, the mission of n-Lorem is personal. Her son Ethan was diagnosed with a nano-rare mutation. A geneticist by training, she joined n-Lorem to oversee …
Q&A #2 with Patient Families
Charissa Lipman joins n-Lorem founder and CEO, and host of the Patient Empowerment Program Podcast, Dr. Stan Crooke, in this question-and-answer episode to discuss additional questions asked during t…
Everybody Lives with Dan Doctoroff
We're diving into the archives to resurface this top downloaded episode from last year. Dan Doctoroff joined Patient Empowerment Program in February 2023 to talk about his mission to support a world …