56 episodes

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.

This podcast is produced by n-Lorem Foundation and hosted by Dr. Stanley T. Crooke, who is our Founder, CEO and Chairman. Our videographer is Jon Magnuson. Our producers are Kira Dineen, Jon Magnuson, Kim Butler, and Amy Williford. To learn more about n-Lorem, visit nlorem.org. Contact us at podcast@nlorem.org.

Patient Empowerment Program: A Rare Disease Podcast n-Lorem Foundation

    • Science
    • 5.0 • 28 Ratings

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.

This podcast is produced by n-Lorem Foundation and hosted by Dr. Stanley T. Crooke, who is our Founder, CEO and Chairman. Our videographer is Jon Magnuson. Our producers are Kira Dineen, Jon Magnuson, Kim Butler, and Amy Williford. To learn more about n-Lorem, visit nlorem.org. Contact us at podcast@nlorem.org.

    Why We Dose Intrathecally

    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) 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 Barriers 
    The Four Tubes – Enteral, Respiratory, Urinary, and the Central Nervous System (CNS) tube 
    How The Body Constructs Barriers 
    What is Cerebrospinal Fluid (CSF) 
    The Blood-Brain Barrier (BBB) 
    The Types of Molecules That Are Admitted to and Excluded From the CNS 
    Intrathecal Administration of Drugs 
    Outwitting the Blood-Brain Barrier 
     
    Survey Link – Patient Empowerment Program Podcast: https://forms.office.com/r/1ik9WNs7QB  
    Register for the 2024 Nano-rare Patient Colloquium: https://www.nlorem.org/nano-rare-patient-colloquium-2024/  

    • 29 min
    Behind the ASO Design and Discovery with Konstantina Skourti-Stathaki, Ph.D.

    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 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 Greece to attend grad school at the University of OxfordAn internship led Nadina to the world of RNALeaving academia to impact livesLessons learned from experiencing drug discovery and developmentThe reason she joined n-LoremDay-to-day activities as Director of ASO Design and discoveryn-Lorem’s internal research management committee to make key decisions regarding programsOngoing research at n-Lorem to enhance allele selectivityStan Crooke on why he pioneered antisense technology

    • 27 min
    Best of the 2023 Nano-rare Patient Colloquium

    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 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 story told by a loved one, and imagine a future where commercial drug discovery organizations can focus on the world’s rarest diseases and more all in this one episode. To access the entire event, visit https://www.nlorem.org/nano-rare-patient-colloquium-2023/
    Register for the 2024 NRPC: https://www.nlorem.org/2024-nano-rare-patient-colloquium-registration-form/

    • 36 min
    The Hurdles of Insuring Patients with Nano-rare Mutations with Alan Lotvin, M.D.

    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 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, conceptualizes these hurdles. 

    • 50 min
    Anna's Story of Hope and Help: FUS-ALS with Sonja Kämpfer and Dr. Neil Shneider

    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 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 daughter’s disease: puzzlement, pain, humility, and now optimism.
    On This Episode We Discuss:
    Anna’s life before her symptoms appearedThe road to a diagnosis and treatment– from Germany to New York CityNeil Shneider and his work with ASOsThe struggles of a family fighting a nano-rare disorderAnna’s mutationAnna’s remarkable progress

    • 43 min
    Mostyn's Story: Mother's Day Special with Zoe Hummel

    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 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 journey to a diagnosisZoe’s advice for parents observing abnormalities in their child's developmentMostyn’s severe epilepsyA road trip to Boston Children’s Hospital in the middle of winterThe ‘unlucky’ diagnosis of KCNB1For-profit companies promising false hope

    • 50 min

Customer Reviews

5.0 out of 5
28 Ratings

28 Ratings

Flygirl3663 ,

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.

Friendly? ,

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.

lalitafer ,

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!

Top Podcasts In Science

Hidden Brain
Hidden Brain, Shankar Vedantam
Radiolab
WNYC Studios
Short Wave
NPR
StarTalk Radio
Neil deGrasse Tyson
Making Sense with Sam Harris
Sam Harris
Ologies with Alie Ward
Alie Ward

You Might Also Like

The Daily
The New York Times
Pod Save America
Crooked Media
The Rachel Maddow Show
Rachel Maddow, MSNBC
The Promised Podcast
TLV1 Studios