Patient Empowerment Program: A Rare Disease Podcast

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

  • 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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Episódios
  • How Cells Phone a Friend: Local Communication
    Mar 19 2025

    Our bodies are like bustling cities of cells, always chatting and working together. They don’t just send long-distance messages to organs—they also gossip locally, getting nearby cells to spring into action. Juxtacrine communication is like a handshake between cells—they have to be up close and personal to pass the message along! Unlike long-distance cellular calls, juxtacrine signaling requires direct contact, where one cell’s surface proteins interact with another’s, triggering a response.

    This episode is brought to you by Hongene Biotech who is continuously innovating to make RNA medicines accessible and affordable to patients worldwide. Visit www.hongene.com

    On this episode we discuss:

    • Paracrine communication
    • Recently found exosomes
    • Cell to cell contact-dependent communication (Juxtacrine signaling)
    • Tunneling microtubes
    • Cell communication conclusions
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    21 minutos
  • How Cells Phone a Friend: Long Distance Communication
    Mar 5 2025

    How do cells communicate with each other? With trillions of cells in the human body, seamless collaboration is essential within this intricate cellular society. Cells work together to form organs, and when one organ needs another to perform a task, they send signals across distances—much like picking up the phone and calling someone. Organ Hotline

    📞 Eyes: Hey, Brain? This is Eyes. We’re reading this post about how organs communicate, and it’s really interesting. 📞 Brain: Oh, show me more! What’s it say? 📞 Eyes: Apparently, organs send signals to each other to get stuff done. Like, the liver calls the stomach when it’s time to detox! 📞 Brain: Whoa, I had no idea they were so organized! Keep reading, I need all the details! 📞 Eyes: I’m on it! But it says they made a whole podcast episode on in. 📞 Brain: Alright, I’m sold! I'm signaling to press play. On This Episode We Discuss:

    • How cells talk to each other
    • Multicellular organisms
    • Why cells create organs
    • Organ to organ communication - Electrochemical
    • Chemical signals - The endocrine system
    • Hormones created by other organs

    This episode is brought to you by Hongene Biotech: https://www.hongene.com/

    Support n-Lorem and nano-rare patient: https://www.nlorem.org/donate/

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    36 minutos
  • Rosie’s Story: A Nano-rare Treatment Pioneer
    Feb 19 2025

    Rosie was the first of three HNRNPH2 patients to receive treatment with an n-Lorem ASO, helping to pave the way for others. Her parents, Nicole and Bobby, share their daughter's journey and how they have adapted as a family to meet her unique needs. As a pediatrician, Nicole understands the complexities of medical care, yet she acknowledges that even she doesn’t always have the answers when it comes to raising a child with a nano-rare disease—a reality that resonates with many nano-rare families. On this episode we discuss:

    2:00 Bobby left his job to help care for Rosie full-time

    7:36 Nicole is a pediatrician and it’s difficult for her to not regularly attend Rosie’s appointments

    11:16 Rosie’s diagnosis of HNRNPH2

    16:00 Even as a pediatrician, Nicole finds nano-rare diseases complex and difficult to navigate

    19:29 What is HNRNPH2?

    26:40 Coming to terms with the diagnosis

    29:35 Weighing the potential risk/benefit and making the decision that Rosie will be the first HNRNPH2 patient treated with an experimental medicine

    37:05 How Rosie is doing after three ASO treatment doses

    40:35 Potential for a placebo effect and allowing multiple observers to notice changes

    Donate to n-Lorem: https://www.nlorem.org/donate/ This episode is brought to you by Hongene Biotech: https://www.hongene.com/

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    50 minutos

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