Episodes
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Your Questions, Answered: 2026 Q&A
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Best of the 2025 Nano-rare Patient Colloquium
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Natacha Gassenbach of Biogen: 2025 Hero of n-Lorem
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Seeing with Grace: A Teen’s Journey with a Rare Vision-Affecting Disease
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Sneak Peak of the 2025 Nano-rare Patient Colloquium
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A Conversation with the 2025 Heroes of n-Lorem
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Meet Rescue 7: Firefighters Serving Patients
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Hope for Kinsley: Facing TUBB4A with Courage and Care
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New Insights into Genetically Caused Developmental Delays
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Research to Advance ASO Technology at n-Lorem
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Commitment to Safety First: The DSMB
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Connor Gooley's Story: A First for TUBB4A Treatment
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What Are iPSCs—and Why Do They Matter?
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Biological Systems & Chemical Networks
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Why ASO Technology?
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Connor Dalby's Story: n-Lorem Patient #001 with Kelley Dalby and Dr. Olivia Kim-McManus
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Our Mission is Personal with Sarah Glass
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How Cells Phone a Friend: Local Communication
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How Cells Phone a Friend: Long Distance Communication
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Rosie’s Story: A Nano-rare Treatment Pioneer
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Sloane's Story: A Shared Medicine
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5-years of n-Lorem: What Have We Learned?
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I Sing the Body Electric
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Q&A - 2024 Nano-rare Patient Colloquium Recap
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Best of the 2024 Nano-rare Patient Colloquium
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Susannah's Progress: Two Years of ASO Treatment with Dr. Jennifer Bain and Luke Rosen
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Sneak Peek of the 2024 Nano-rare Patient Colloquium
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The Building Blocks of What's Possible with David Butler, Ph.D.
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Knowledge is Power: Part 2
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Knowledge is Power: Part 1
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Adopting a Rare Son with Paul Compton
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Ion Channel Mutations: The Complexities of Treatment
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Why We Dose Intrathecally
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Behind the ASO Design and Discovery with Konstantina Skourti-Stathaki, Ph.D.
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Best of the 2023 Nano-rare Patient Colloquium
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The Hurdles of Insuring Patients with Nano-rare Mutations with Alan Lotvin, M.D.
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Anna’s Story of Hope and Help: FUS-ALS with Sonja Kämpfer and Dr. Neil Shneider
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Mostyn’s Story: Mother’s Day Special with Zoe Hummel
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Physicians and Institutions Transform the Lives of Nano-rare Patients with Olivia Kim-McManus, M.D.
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Advanced Genetics Part 2
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Advanced Genetics Part 1
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Sharing Hope Through Storytelling & Connection with Shanna Tolbert
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Our Mission is Personal with Sarah Glass
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Q&A #2 with Patient Families
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Everybody Lives with Dan Doctoroff
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Best of the 2023 Nano-rare Patient Colloquium
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How to Think About ”Risk” Part 2
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How to Think About ”Risk” Part 1
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Inheriting Huntington’s Disease and Aspiring to Prolong Lives with Jeff Carroll
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Living a Longer, Healthier, and More Fun Life with Chip Wilson