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Patient Empowerment Program: A Rare Disease Podcast

Patient Empowerment Program: A Rare Disease Podcast

Auteur(s): n-Lorem Foundation (Dr. Stan Crooke Amy Williford Kim Butler Andrew Serrano Jon Magnuson and Kira Dineen)
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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. Crroke, who is the 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.Copyright 2024 n-Lorem Foundation Hygiène et mode de vie sain Science Sciences biologiques Troubles et maladies
Épisodes
  • Where Emerging FDA Draft Guidance may Lead for Nano-rare Patients
    Mar 4 2026

    On This episode We Discuss:

    2:30 – Andrew realized that finance plays a very large role in drug development and is an obstacle in for rare disease patients getting the treatments they need

    4:30 – The triple whammy of drug development

    6:22 – Economic decisions when lives are at stake; Balancing the ethical considerations of medicine and finance

    12:17 – Small biotech companies are better for innovation, and a distributed investment process is more productive; Innovation comes from orthogonal thinking

    22:40 – Very small prevalence disease drug programs tend to get terminated at commercial organizations; Movement toward finding a way to create a commercial path for extremely rare disease drugs

    25:45 – FDA draft guidance for potential commercialization of nano-rare medicines; Where the guidance may lead; Value of a statistical life

    30:32 – The cost of a nano-rare patient’s care; Courageous moves of the FDA

    35:00 – Regulatory processes merging with the advances of science while still protecting patients

    Andrew W. Lo is the Charles E. and Susan T. Harris Professor at the MIT Sloan School of Management, director of MIT’s Laboratory for Financial Engineering, and principal investigator at MIT’s Computer Science and Artificial Intelligence Laboratory. His healthcare-related research interests include: new financial engineering tools and business models for drug and device development and healthcare delivery, especially for rare and ultra-rare diseases; statistical methods for incorporating patient preferences into the drug approval process; predicting clinical trial outcomes via machine learning techniques; and novel reimbursement models for creating a robust gene and cell therapy ecosystem. He is a co-founder of BridgeBio Pharma, QLS Advisors, Quantile Health, and Uncommon Cures; a director of AbCellera, Atomwise, BridgeBio, Uncommon Cures, and Vesalius; and a member of the advisory board to the American Cancer Society’s BrightEdge Impact Fund. Lo received his B.A. in economics from Yale University and his A.M. and Ph.D. in economics from Harvard University.

    Patient Empowerment Program host: Stan Crooke, n-Lorem Founder and CEO

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    43 min
  • Reflections from the n-Lorem Chairman | 2026
    Feb 11 2026

    In this video message, Chairman Stan Crooke reflects on the close of 2025, six years after founding n-Lorem. He shares his gratitude for the community that made it the organization’s most successful year to date and offers perspective on how progress across every area of the foundation is carrying n-Lorem forward into 2026.

    Read the 2026 Chairman Letter

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    18 min
  • Miracles of Science: Genomics
    Feb 4 2026

    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 can only be described as miracles of science. Not the fairytale kind. Not wishes granted overnight. These miracles are earned by humans. Built over centuries by scientists whose discoveries stacked, evolved, and refined over time.

    They are the result of human curiosity, persistence, and an ever-deepening understanding of biology, and they’ve fundamentally changed how we see the world and treat disease.

    Today’s miracle: Genomics.

    Coming next: iPSCs, the RNA World, and Antisense Technology. The breakthroughs that pushed possibility even further.

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    The Patient Empowerment Program podcast is hosted by n-Lorem Founder and CEO, Stan Crooke. Dr. Crooke recently joined the advisory board of the CNBC Cures, an initiative bringing together families, doctors, investors and regulators with one goal in mind: helping to raise awareness of, and improve patient outcomes for, the 30 million Americans suffering from rare diseases. You can join the CNBC Cures Newsletter here: https://www.cnbc.com/cnbc-cures-newsletter/

    Today's sponsor is ChemGenes: https://www.chemgenes.com/

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    33 min
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