Episodes
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Sneak Peek of the 2026 Nano-rare Patient Colloquium
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Realities of the Nano-rare: Siblings, Unfairness and Hard Truths with Sally Jackson
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Silencing ALS with Neil Shneider
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Miracles of Science: Antisense Technology
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The Personal Rare Disease Journey Behind CNBC Cures with Becky Quick
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Patient Story: Understanding ALS with Bill O'Sullivan and Neil Shneider, M.D., Ph.D.
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Miracles of Science #3: RNA World Part 3
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Miracles of Science #3: RNA World Part 2
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Miracles of Science #3: RNA World Part 1
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Realizing Hope for Layken
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Realities of the Nano-rare: Episode 2 Oliver Glass
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Realities of the Nano-rare: Episode 1 with Sarah Glass
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Miracles of Science #2: Induced Pluripotent Stem Cells
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Where Emerging FDA Draft Guidance may Lead for Nano-rare Patients
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Reflections from the n-Lorem Chairman | 2026
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Miracles of Science: Genomics
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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.