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)
Listen for free

  • 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
    Show More Show Less
Biological Sciences Hygiene & Healthy Living Physical Illness & Disease
Show More Show Less
Episodes
  • Susannah's Progress: Two Years of ASO Treatment with Dr. Jennifer Bain and Luke Rosen

    Susannah's Progress: Two Years of ASO Treatment with Dr. Jennifer Bain and Luke Rosen
    Oct 16 2024

    Two years of treatment—two years of strength, courage, and blazing a trail for other nano-rare patients! 🎉 Susannah's story was the first shared on the Patient Empowerment Program podcast. Now, two years later, her father, Luke Rosen, and her physician, Dr. Jennifer Bain, detail Susannah’s remarkable progress since beginning regular treatments—highlighting improvements in her motor skills, cognition, energy, and more!

    Susannah's Story (May 25, 2022)

    Donate

    2024 Nano-rare Patient Colloquium

    On This Episode We Discuss:

    • 3:10 Susannah's KIF1A mutation
    • 5:50 Luke and his wife Sally changed their careers to help find a treatment for KIF1A
    • 7:23 How Susannah's family first heard of n-Lorem
    • 8:43 Jen on the Columbia University Medical Center team and the decision to treat Susannah
    • 12:25 Luke's experience bringing his daughter to receive an experimental ASO treatment for the first time
    • 14:30 The positive outcomes and observations of Susannah's treatment
    • 26:15 What Dr. Bain has learned since treating Susannah
    • 29:15 Luke on the impact and importance of the little things
    • 31:15 Advice to other parents
    • 35:40 Being part of a community of nano-rare patients and families
    Show More Show Less
    41 mins
  • Sneak Peek of the 2024 Nano-rare Patient Colloquium

    Sneak Peek of the 2024 Nano-rare Patient Colloquium
    Oct 2 2024

    Take a glimpse behind the curtain as we offer you a sneak peek of the 2024 Nano-rare Patient Colloquium. We’re thrilled to welcome back longtime biotech journalist and current Features Editor of The Transmitter, Brady Huggett, to the podcast. In an interview with n-Lorem founder and CEO Stan Crooke, they preview the upcoming Colloquium, diving into key topics that will shape the conversation at the event and around nano-rare diseases in the year ahead. On This Episode We Discuss:

    2:21 How many Investigational New Drugs (INDs) has n-Lorem filed?

    5:22 How many patients treated with an n-Lorem ASO have been on therapy long enough to observe a benefit?

    6:50 What does it mean for a patient like Susannah who has experienced a significant decrease in behavioral arrest incidents since receiving treatment

    9:39 Providing ASOs to patients earlier

    13:25 INDs for the ‘n-of-few'

    23:15 Introducing Whole Genome Sequencing (WGS) for all newborns

    29:05 Stan’s perspective on leadership

    33:50 n-Lorem's goals for the 2024 Nano-rare Patient Colloquium

    Register for the 2024 Nano-rare Patient Colloquium Donate

    Biogen.com
    Show More Show Less
    42 mins
  • The Building Blocks of What's Possible with David Butler, Ph.D.

    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/
    Show More Show Less
    30 mins

What listeners say about Patient Empowerment Program: A Rare Disease Podcast

Average Customer Ratings

Reviews - Please select the tabs below to change the source of reviews.

Audible.com.au reviews

Amazon Reviews
No Reviews are Available
In the spirit of reconciliation, Audible acknowledges the Traditional Custodians of country throughout Australia and their connections to land, sea and community. We pay our respect to their elders past and present and extend that respect to all Aboriginal and Torres Strait Islander peoples today.