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)
  • Resumen

  • 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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Episodios
  • Our Mission is Personal with Sarah Glass
    Apr 2 2025

    For n-Lorem's Chief Operating Officer, Sarah Glass, the mission of n-Lorem is deeply personal. Her son Ethan was diagnosed with a nano-rare mutation, a journey that has shaped her commitment to the cause. A geneticist by training, Sarah joined n-Lorem to help lead and guide the organization in its efforts to offer hope and potential help through treatment opportunities to nano-rare patients and their families. This is more than just a job for her—she is driven by her love for her son and a passion to serve the entire nano-rare community. This episode is proudly sponsored by Hongene Biotech.

    “We are changing the landscape of rare disease drug discovery and development – starting with the most rare.” – Sarah Glass, Ph.D.

    On This Episode We Discuss:

    ✔️ Sarah’s background in science and her early interests

    ✔️ How working at a Contract Research Organization (CRO) shaped her understanding of the patient experience

    ✔️ The challenges of rare disease clinical trials and why they are still relatively new

    ✔️ Sarah’s son, Ethan, and his journey as a nano-rare patient

    ✔️ How long it took for Ethan to receive a diagnosis and the symptoms he faces

    ✔️ How Sarah discovered n-Lorem, a nonprofit providing free, lifetime treatment for nano-rare patients

    ✔️ The emotional journey of caring for a nano-rare child—how parents navigate hope and uncertainty

    ✔️ What Sarah has learned while working at n-Lorem

    ✔️ The biggest surprises in her journey as both a mother and a scientist

    If you’re passionate about rare diseases, personalized medicine, and patient advocacy, this episode is a must-watch! 💙

    🔔 Subscribe for more stories from the rare disease community! #RareDisease #NanoRare #nLorem #PatientAdvocacy #geneticdisorders

    Donate to n-Lorem: www.nlorem.org/donate

    More about Hongene: www.hongene.com

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    42 m
  • 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 m
  • 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 m

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