All Episodes

Miracles of Science: Antisense Technology

The Personal Rare Disease Journey Behind CNBC Cures with Becky Quick

Patient Story: Understanding ALS with Bill O'Sullivan and Neil Shneider, M.D., Ph.D.

Miracles of Science #3: RNA World Part 3

Miracles of Science #3: RNA World Part 2

Miracles of Science #3: RNA World Part 1

Realizing Hope for Layken

Realities of the Nano-rare: Episode 2 Oliver Glass

Realities of the Nano-rare: Episode 1 with Sarah Glass

Miracles of Science #2: Induced Pluripotent Stem Cells

Where Emerging FDA Draft Guidance may Lead for Nano-rare Patients

Reflections from the n-Lorem Chairman | 2026

Miracles of Science: Genomics

Your Questions, Answered: 2026 Q&A

Best of the 2025 Nano-rare Patient Colloquium

Natacha Gassenbach of Biogen: 2025 Hero of n-Lorem

Seeing with Grace: A Teen’s Journey with a Rare Vision-Affecting Disease

Sneak Peak of the 2025 Nano-rare Patient Colloquium

A Conversation with the 2025 Heroes of n-Lorem

Meet Rescue 7: Firefighters Serving Patients

Hope for Kinsley: Facing TUBB4A with Courage and Care

New Insights into Genetically Caused Developmental Delays

Research to Advance ASO Technology at n-Lorem

Commitment to Safety First: The DSMB

Connor Gooley's Story: A First for TUBB4A Treatment

What Are iPSCs—and Why Do They Matter?

Biological Systems & Chemical Networks

Why ASO Technology?

Connor Dalby's Story: n-Lorem Patient #001 with Kelley Dalby and Dr. Olivia Kim-McManus

Our Mission is Personal with Sarah Glass

How Cells Phone a Friend: Local Communication

How Cells Phone a Friend: Long Distance Communication

Rosie’s Story: A Nano-rare Treatment Pioneer

Sloane's Story: A Shared Medicine

5-years of n-Lorem: What Have We Learned?

I Sing the Body Electric

Q&A - 2024 Nano-rare Patient Colloquium Recap

Best of the 2024 Nano-rare Patient Colloquium

Susannah's Progress: Two Years of ASO Treatment with Dr. Jennifer Bain and Luke Rosen

Sneak Peek of the 2024 Nano-rare Patient Colloquium

The Building Blocks of What's Possible with David Butler, Ph.D.

Knowledge is Power: Part 2

Knowledge is Power: Part 1

Adopting a Rare Son with Paul Compton

Ion Channel Mutations: The Complexities of Treatment

Why We Dose Intrathecally

Behind the ASO Design and Discovery with Konstantina Skourti-Stathaki, Ph.D.

Best of the 2023 Nano-rare Patient Colloquium

The Hurdles of Insuring Patients with Nano-rare Mutations with Alan Lotvin, M.D.

Anna’s Story of Hope and Help: FUS-ALS with Sonja Kämpfer and Dr. Neil Shneider

Mostyn’s Story: Mother’s Day Special with Zoe Hummel

Physicians and Institutions Transform the Lives of Nano-rare Patients with Olivia Kim-McManus, M.D.

Advanced Genetics Part 2

Advanced Genetics Part 1

Sharing Hope Through Storytelling & Connection with Shanna Tolbert

Our Mission is Personal with Sarah Glass

Q&A #2 with Patient Families

Everybody Lives with Dan Doctoroff

Best of the 2023 Nano-rare Patient Colloquium

How to Think About ”Risk” Part 2

How to Think About ”Risk” Part 1

Inheriting Huntington’s Disease and Aspiring to Prolong Lives with Jeff Carroll

Living a Longer, Healthier, and More Fun Life with Chip Wilson

Autopilot: The Autonomic Nervous System

Better Health, Brighter Future for Rare with Dan Curran

Sneak Peek of the 2023 Nano-rare Patient Colloquium

Liver: Diseases

Liver: The Master Chemist

Liver: Metabolic Magic

Bottling Hope

Pursuing Diagnoses and Sequencing

Lungs: Heavy Breathing — Diseases and Obstructions

Lungs: Defending Airways

One Year Anniversary: It Takes a Rare Community

Coming Together for the Nano-rare Patient

Q&A #1 with Patient Families

Bits and Bytes Help Streamline Bench to Bedside

Fostering Collaboration to Fight ALS

The Energy of Life

Everybody Lives

Cardiovascular System Part 4B: (The Spigot) Kidney Diseases

Cardiovascular System Part 4A: (The Spigot) Kidney Functions

Cardiovascular System Part 3: (The Pipes) Arteries and Veins

CROs and Their Role for Nano-rare

Cardiovascular System Part 2B: (The Pump) Heart Diseases

Cardiovascular System Part 2A: (The Pump) Heart Anatomy

Shifting Mindsets to Expedite Rare Treatments

Cardiovascular System Part 1: Blood

Coming Together for the Nano-rare Patient

Antisense (How We Do It at n-Lorem)

Anna’s Story of Hope and Help

Why ASO Technology

Advocating for Rare Patients

Drug Discovery Platforms

Clinical Trials for One Patient

How Drugs are Used

A Diagnostic Odyssey: The UDN and n-Lorem’s Roles

Biological Systems & Chemical Networks

What is n-Lorem?

Susannah’s Story with Dr. Wendy Chung and Luke Rosen

Patient Empowerment Program