Patient Empowerment Program: A Rare Disease Podcast

By: n-Lorem Foundation (Dr. Stan Crooke Amy Williford Kim Butler Andrew Serrano Jon Magnuson and Kira Dineen)
  • Summary

  • 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
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Episodes
  • The Building Blocks of What's Possible with David Butler, Ph.D.
    Sep 18 2024

    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 Hongene

    12:00 Hongene is a strong supporter of n-Lorem. Why?

    15:30 David’s origins in the chemistry field

    17:38 Incremental progress that has been made in science and manufacturing

    Links:

    2024 Colloquium - https://www.nlorem.org/nano-rare-patient-colloquium-2024/

    Make Hope Possible with a Donation - https://www.nlorem.org/donate/

    Hongene - https://www.hongene.com/

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    30 mins
  • Knowledge is Power: Part 2
    Sep 3 2024
    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 teach us about health and disease and opportunities to treat more common diseases
    • What are all the functions of human genes?
    • Are there gene products that have redundant functions?
    • Do identical mutations cause identical phenotypes?
    • n-Lorem can demonstrate that ASOs work on general types of disease challenges
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    30 mins
  • Knowledge is Power: Part 1
    Aug 21 2024

    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 Colloquium

    On This Episode We Discuss:

    • Two most common words in medicine: Health and Disease
    • The Scientific Method
    • The Importance of Single Variables
    • Orthogonal Thinking
    • We Are at a Unique Moment in Medical History
    • Genomics
    • Advances in Omics
    • Biological Networks and AI
    • Facile Collection, Maintenance, Growth and Differentiation in the Lab
    • Antisense Technology
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    35 mins

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