The Ultimate Family Meeting Preview | What Not to Miss, Featured Guests & How to Join From Anywhere

The Ultimate Family Meeting Preview | What Not to Miss, Featured Guests & How to Join From Anywhere

Spotlight Family Social Media Account: Abilities First Texas, by Katy Pipak Williams, Mom to EK who has AHChttps://www.instagram.com/abilities.first.tx/# FAMILY MEETING INFO:Virtual attendance is available again this year. Virtual attendees will be able to watch presentations live and participate in live Q&A sessions throughout the weekend.Virtual registration is $50. If cost is a barrier, email Izzie for help.Time zone conversions for live sessions:• California — 6:00 AM PDT• Chicago — 8:00 AM CDT• London — 2:00 PM BST• Central Europe — 3:00 PM CEST• Dubai — 5:00 PM GST• São Paulo — 10:00 AM BRT• China — 9:00 PM CST• Australia (Sydney) — 11:00 PM AESTYou can view the full agenda here: https://static1.squarespace.com/static/5bdfc7a3cef372e7bbfcb463/t/6a1dadd2ab0f493a33ec674c/1780329962252/Meeting+Agenda To register for virtual attendance: https://cureahc.charityproud.org/EventRegistration/Index/26166?fbclid=IwY2xjawQDFAZleHRuA2FlbQIxMABicmlkETIxakZpaXcwNlpHYTlNcG9sc3J0YwZhcHBfaWQQMjIyMDM5MTc4ODIwMDg5MgABHk_R6S8mJqHLSKAgXN1oS8tIiy4eL-nAoAcXtl3olG7BWlhvlJOieC_MC00X_aem_2y3PUqrvGL760Vnv2ykyFQNon-English Speakers: We will release recordings after the meeting along with transcripts in various languages.Research Reminders:1. Patient Registry: https://docs.google.com/forms/d/e/1FAIpQLSc8X-QGp9E4Hj-kIZFIly_Nefu9sWt7k3vdoZToNcRXPsGsUA/viewform2. Rare Hope Patient Surveys: https://www.rare-hope.org/ahc-research-program/patient-data3. Patient Priorities Survey: https://docs.google.com/forms/d/e/1FAIpQLSfbrkVDvQz8M2-pvfJq0rmJqbbnOVzhGOPApdTOke02opeAJQ/viewform4. Sleep, Triggers, Medications, and Headaches: Insight into the AHC Patient Experience: https://docs.google.com/forms/d/e/1FAIpQLSfWsLKeUi9Lm5TU1KLg9FsfyHuZ-q47-9xzy7n43bt1vQ9weg/viewform5. In July 2026 -- stay tuned to be able to start a Combined Brain account to begin enrolling in patient-driven data collection in order to have your patient’s information included in ongoing research efforts Podcast Submission link: https://forms.gle/gU31JB8GAfSnmWWL6-Submit to have your social media account featured-Submit to appear on the podcast and share your storyJoin our global AHC Research & Support Group All AHC patients, ATP1A3 and ATP1A2 related conditions, all phenotypes and genotypes welcome! https://www.facebook.com/share/g/18Ztn3AiGf/ Need support? Contact Izzie at izzie@cureahc.org, on Facebook at @IzzieDuval or WhatsApp!Podcast Team:Izzie Duval - HostHannah Zimmer - Co-host, Producer & Social MediaCorey Zimmer - Producer & Editor

Hannah & Archie’s Story | AHC, Epilepsy + Rewriting Expectations Around What Matters Most

Welcome to Behind the Episode, a rare disease advocacy podcast by Cure AHC.  This podcast is about the families, the people, the science, the advocacy, the stories, the hope, and the strength that exists behind the AHC episodes. Behind the Episode is meant to be a living resource for families—new and seasoned alike.  This month we are thrilled to share our first Family Diagnosis episode, featuring Hannah Zimmer discussing her son Archie’s diagnosis story, epilepsy, life with two under two, and how re-imagining priorities to quality of life has helped their family process and move forward with joy. Hannah & Archie’s accounts: Facebook: Archie's Anchors [https://www.facebook.com/profile.php?id=61573580930442] Instagram: Archie's Anchors [https://www.instagram.com/archiesanchors?utm_source=ig_web_button_share_sheet&igsh=ZDNlZDc0MzIxNw==] www.archiesanchors.org [http://www.archiesanchors.org] Family Social Media Spotlight: Adventure with Jett TikTok: adventure.with.jett [https://l.facebook.com/l.php?u=https%3A%2F%2Ftiktok.com%2F%40adventure.with.jett%3Ffbclid%3DIwZXh0bgNhZW0CMTAAYnJpZBExeXhiSVZqOWRLWGJReVVYNXNydGMGYXBwX2lkEDIyMjAzOTE3ODgyMDA4OTIAAR4-hQIQbm0uwMggR-thad0cAxP5YWQY9X5iFCGq7lh5NJ3xhdxFQwkkN8jGZQ_aem_qIhZuHZMQ1n60s5YGKG_KQ&h=AT6dl-tO3DsFKttb3jmIySf80GyrNU2PmC5w-aJZmX1bzWceEhIsk51YH9KGrDNTO2iKy2nzCxqKDs559kye-QsEZclF6485PEgP2SX1N5BTVWstHvWnY08wpjVlyCuqu6vxPV4] YouTube: adventurewithjett [https://l.facebook.com/l.php?u=https%3A%2F%2Fyoutube.com%2F%40adventurewithjett%3Ffbclid%3DIwZXh0bgNhZW0CMTAAYnJpZBExeXhiSVZqOWRLWGJReVVYNXNydGMGYXBwX2lkEDIyMjAzOTE3ODgyMDA4OTIAAR7WRz_zrLSpxpNgEnNxX4RzhJy2IvYh_W3hl8Wt6K6fYIkzrg35DiokK4ULhg_aem_qSwwExlrKd5KDt5LHYGgPQ&h=AT6LBRDFueIdwTdNdxHeQ_OoNDIqh9u0-39Z1PRFcXFP_Nt3VbEx5US8raRpb4ehFHfrKrUJsEjt0O2aFCLzszB5kUsVmYHrnfdL1RX7cEMhAZU_oYReBaX8DtPv9hmYC5X8SXE] Facebook: Adventure with Jett [https://www.facebook.com/profile.php?id=61587899072219] Instagram: Adventure with Jett [https://www.instagram.com/adventurewithjett?utm_source=ig_web_button_share_sheet&igsh=ZDNlZDc0MzIxNw==] Facebook Group for any AHC, ATP1A2, ATP1A3 patients, and related disorder families: JOIN HERE [https://www.facebook.com/share/g/16u33LM8Gq/] We can’t wait to feature more AHC & related disorder families’ stories! If you are interested in being featured on Behind the Episode use the link HERE [https://forms.gle/LmzZ7gKUBmwPacaV8]. ———— Past Action Items: AHC Research Survey links: Patient Priorities: Which AHC Symptoms are the Most Critical Targets for Therapy?  [https://docs.google.com/forms/d/e/1FAIpQLSfbrkVDvQz8M2-pvfJq0rmJqbbnOVzhGOPApdTOke02opeAJQ/viewform?pli=1%0A%5C] Sleep, Triggers, Medications, and Headaches: Insight into the AHC Patient Experience [https://docs.google.com/forms/d/e/1FAIpQLSfWsLKeUi9Lm5TU1KLg9FsfyHuZ-q47-9xzy7n43bt1vQ9weg/viewform] To enroll in the AHC Patient Registry (Rare Hope) [https://docs.google.com/forms/d/e/1FAIpQLSc8X-QGp9E4Hj-kIZFIly_Nefu9sWt7k3vdoZToNcRXPsGsUA/viewform] Behind the Episode Podcast Team: Izzie Duval - Host  Hannah Zimmer - Producer & Co-Host Corey Zimmer - Editor & Producer To contact us: izzie@cureahc.org [izzie@cureahc.org],  hannah@cureahc.org [Hannah@cureahc.org],  archiesanchors@gmail.com [archiesanchors@gmail.com]

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 [https://www.rare-hope.org/news/first-neuro-breakthrough-next-generation-genetic-editing-tool-rescues-devastating-childhood-brain-disease-vivo] More about Nina and Simon Frost: https://www.rare-hope.org/who-we-are/our-founders [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 [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 [https://www.rare-hope.org/impact]  Rare Hope in the News: https://www.rare-hope.org/news [https://www.rare-hope.org/news] Full Rare Hope Promo video: https://youtu.be/XatOliAjryM [https://youtu.be/XatOliAjryM] ACTION ITEMS: AHC Research Survey links: Patient Priorities: Which AHC Symptoms are the Most Critical Targets for Therapy?  [https://docs.google.com/forms/d/e/1FAIpQLSfbrkVDvQz8M2-pvfJq0rmJqbbnOVzhGOPApdTOke02opeAJQ/viewform?pli=1%0A%5C] Sleep, Triggers, Medications, and Headaches: Insight into the AHC Patient Experience [https://docs.google.com/forms/d/e/1FAIpQLSfWsLKeUi9Lm5TU1KLg9FsfyHuZ-q47-9xzy7n43bt1vQ9weg/viewform] To enroll in the AHC Patient Registry (Rare Hope): https://docs.google.com/forms/d/e/1FAIpQLSc8X-QGp9E4Hj-kIZFIly_Nefu9sWt7k3vdoZToNcRXPsGsUA/viewform [https://docs.google.com/forms/d/e/1FAIpQLSc8X-QGp9E4Hj-kIZFIly_Nefu9sWt7k3vdoZToNcRXPsGsUA/viewform]  Human Timebombs, AHC Documentary  https://humantimebombs.com/ [https://humantimebombs.com/]  Baby KJ : https://youtu.be/mUQWWLErLaY [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 [izzie@cureahc.org], hannah@cureahc.org [Hannah@cureahc.org], archiesanchors@gmail.com [archiesanchors@gmail.com]

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.