Behind the Episode: An Advocacy Podcast by Cure AHC

Cure AHC
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  • UPDATED MONTHLY

A podcast created for the AHC community, by the AHC community. Bringing you information that us hopeful, digestible, and supportive! Each episode will take you behind the moments of life with AHC, sharing research updates, insights from specialists, guidance on patient care, stories and support for families walking this rare disease journey.

Episodes

  1. 3D AGO

    Behind the Episode: Ep 2 w/ Nina and Simon Frost

    Welcome to Behind the Episode, a rare disease advocacy podcast by Cure AHC. Months ago, we gathered your questions as a community — and this conversation is where those answers live. This is a longer episode, because we didn’t want to cut any of the information that feels so important and so hopeful for families. But we also know your time is limited, so we’ve added chapters and time stamps in the show notes so you can easily jump to the sections you need. The recent prime-editing success in the AHC mouse model is the first time a neurological or central nervous system disease has ever been successfully genetically edited in a living animal. Not in a dish.Not in isolated cells.In a living mammal. We’ve seen CRISPR breakthroughs in the eye, the liver, heart and blood — you may have heard about Baby KJ, or the FDA-approved treatments for sickle cell disease and metabolic liver disorders… But crossing the blood–brain barrier and successfully editing a neurological disease in vivo has never happened before. Until now. You’ll find links in the show notes to the key action steps from today’s episode and more information about guests Nina and Simon Frost, Rare Hope and other relevant resources. **DISCLAIMER**In reference to the mouse model being the first ever editing success in a living mammal - this was the first ever PRIME editing success. Base editing success has been shown for a number of years. 3:43 - Rare Hope Intro 7:25 - Opening Discussion 26:44 - The research process and Nina's role 34:25 - Development process 39:02 - Intro to gene editing, treatments, and analogies 48:40 - Community question: Is anyone working on genetic treatments for AHC? 53:37 - What do we hope to change? Does age factor in? 57:14 - Could genetic based epilepsy be improved through gene therapy? 59:43 - When could we see human clinical trials? 1:06:23 - AHC research being recognized 1:09:26 - Engaging patients in research 1:17:10 - Do we need to raise millions to give our kids access to treatments? 1:24:05 - Is there an age limit? 1:29:54 - What gives you the most hope? 1:35:38 - Optimism moving forward First-in-Neuro Breakthrough: Next-Generation Genetic Editing Tool Rescues Devastating Childhood Brain Disease in vivo More about Nina and Simon Frost: https://www.rare-hope.org/who-we-are/our-founders  More about the AHC Research Program: https://www.rare-hope.org/ahc-research-program/about-the-ahc-research-program  More about Rare Hope’s Impact: https://www.rare-hope.org/impact  Rare Hope in the News: https://www.rare-hope.org/news Full Rare Hope Promo video: https://youtu.be/XatOliAjryM ACTION ITEMS: AHC Research Survey links: Patient Priorities: Which AHC Symptoms are the Most Critical Targets for Therapy?  Sleep, Triggers, Medications, and Headaches: Insight into the AHC Patient Experience To enroll in the AHC Patient Registry (Rare Hope): https://docs.google.com/forms/d/e/1FAIpQLSc8X-QGp9E4Hj-kIZFIly_Nefu9sWt7k3vdoZToNcRXPsGsUA/viewform  Human Timebombs, AHC Documentary  https://humantimebombs.com/  Baby KJ : https://youtu.be/mUQWWLErLaY Behind the Episode Podcast Team: Izzie Duval, Host  Hannah Zimmer - Producer & Co-Host Corey Zimmer - Editor & Producer To contact us: izzie@cureahc.org, hannah@cureahc.org, archiesanchors@gmail.com

    1h 42m
  2. Behind the Episode: Ep 1 w/ Simon Frost and Stephen Henderson

    FEB 1

    Behind the Episode: Ep 1 w/ Simon Frost and Stephen Henderson

    In Episode 1, host Izzie Duval speaks with former Cure AHC President and RARE Hope co-founder Simon Frost and newly elected Cure AHC President Stephen Henderson about how their children’s AHC diagnoses sparked their journeys into advocacy, leadership, and building momentum toward new breakthroughs in AHC research.

    55 min
  • 2 Episodes

About

A podcast created for the AHC community, by the AHC community. Bringing you information that us hopeful, digestible, and supportive! Each episode will take you behind the moments of life with AHC, sharing research updates, insights from specialists, guidance on patient care, stories and support for families walking this rare disease journey.

Information