Patient Empowerment Program: A Rare Disease Podcast

Join Stan Crooke, n-Lorem founder, CEO, and host of the Patient Empowerment Program Podcast, alongside Amy Williford, Sr. Director of Communications and Donor Relations, for a special episode. In this post-colloquium Q&A, Stan recaps the 2024 Nano-rare Patient Colloquium and the progress n-Lorem has made as the organization nears its 5-year anniversary, shares his candid reflections on the event, and dives into additional thoughts and questions that weren’t covered. Do you have additional questions? Email [email protected].   Question Bank: 2:15 How do you think the event went this year? 6:05 Were observations of benefit expected in patients so quickly?  9:47 What do you expect the number of patients treated to be next year? 10:57 Do you have a message to supporters? 12:30 Were there any disappointments from the colloquium? 13:21 What are the most important things that n-Lorem has learned this year?  16:13 Do you think n-Lorem’s processes will be outlined every year at the event? 18:05 How is AI incorporated at n-Lorem? 19:10 Does your recently announced deal with GondolaBio reflect the sustainability strategy discussed at the event? 20:53 n-Lorem is celebrating 5 years in 2025. Did you expect the foundation to be where it is today? 21:28 Does n-Lorem plan to expand into Europe and elsewhere?   Make hope possible with a donation.

This year’s Colloquium spanned two perfect autumn days in beautiful Cambridge, Massachusetts where hundreds of nano-rare community members from around the world gathered under a single roof to learn, connect, and support one another. We’re pleased to announce that the event welcomed over 750 attendees, both in-person and virtually. It’s each and every one of you who made the event such a success! For this special episode, we gathered the ‘Best’ clips from the event for you to learn from and enjoy. To submit questions for our upcoming Q&A episode, email [email protected]. We can’t wait to hear from you! 2024 Colloquium Recap On this episode: 2:07 Daphne Graskewicz-Prado, ASO The Perilous Journey to Diagnosis and Treatment for Nano-rare 9:35 Ryan Strankowski and Dr, Nelson Leung, Ryan’s Journey and Clinical Experience 15:55 Luke Rosen, Megan Hedstrom, and Dr. Jennifer Bain, Susannah’s & Sloane’s Journeys and Unique Clinical Experiences 21:49 Kelley Dalby and Dr. Olivia Kim-McManus, Connor’s Journey and Clinical Experience 30:00 Dr. Julie Ziobro and Dr. Horacio Kaufmann, Building a Nano-rare Network and Managing Institutional Challenges 37:38 Dr. Andrew Lo and Dr. Alan Lotvin, Innovative Ways to Support Nano-rare 42:30 Dr. Toby Ferguson, Dr. Liz Berry-Kravis, and Dr. Eugene Shneider, Changing the World One Patient at a Time 47:30 Dr. Kate Dawson, What It Means to be a Parter with n-Lorem for Nano-rare 50:23 Dr. Stanley Crooke, Providing Hope for a Better Future, One Nano-rare Patient at a Time

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

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