All episodes
Developing a New Class of Therapies Based on a Natural Cargo Carrier
Reaching Beyond the Limits of Enzyme Replacement Therapies with Gene Therapies
One Woman’s Journey as a Caregiver to a Husband with Frontotemporal Dementia
Treating Rare Endocrine Disorders with Therapeutic Peptides
Tackling the Pricing Challenges for Advanced Therapies for Rare Diseases
Addressing the Current Limitations of AAV Gene Therapies
Examining the Legislative Landscape for Rare Disease Drug Development
How a Drug Setback Became a Patient Community’s Gain
Helping Regulators and Drug Developers Understand the Challenges of Living with Fabry Disease
Addressing Racial Disparities in a Rare Blood Cancer
How New Sequencing Technology Promises to Alter the Diagnostic Odyssey
Leveraging Technology to Empower Patients and Decentralize Clinical Trials
Equipping The Next-Generation Rare Disease Patient Advocate
How a Family Raced from Diagnosis to Experimental Gene Therapy in Three Years
How One Advocate Went from Losing His Hearing to Being Heard
Building a Pipeline of Therapies to Treat Rare Mineralization Disorders
Advancing an Oral Alternative to Infused and Injected Therapies for HAE
Embracing the Promise of Patient-Centered Biotechnology
Learning to Live with Loss
Using Cryptocurrency to Tap into the Wisdom of Crowds
A Therapy for a Rare Neurodegenerative Disease Moves Towards Regulatory Review
Transforming the Treatment of Neuromuscular Diseases with Next-Gen Oligonucleotides
Teaching the Immune System to Let Medicines Do Their Job
Designing Clinical Trials with the Patient in Mind
Keeping Clinical Trials Running Smoothly
A Vision for Patient-Centric Gene Therapy Development
Using Genetic Testing to Address Disparities in Care for Kidney Disease
Why Reforms to the Accelerated Approval Pathway Threaten Rare Disease Drug Development
A Longstanding Academic-Nonprofit Collaboration Gives Rise to an ALS Drug Company
Targeting Regulatory RNA to Upregulate Gene Expression to Treat Rare Diseases
Developing a New Approach to Treat Rare, Autoimmune Conditions
Bridging the Gap Between Basic and Commercial Research for Rare Disease
Stoking Functional Copies of Genes to Compensate for Mutated Ones
Targeting ALS with Synergistic Combinations of Therapies
How a Polish Rare Disease Organizations Is Helping Its Ukrainian Neighbors
Powering a New Era of Genetic Medicine
Searching for a Diagnosis
A Novel Way to Deliver Rare Disease Therapies
Bringing Diversity, Equity, and Inclusion to the Books We Read
From Mila to Millions: Scaling N of 1 Therapies
Genetically Modifying Patients’ Skin Cells to Treat Rare Diseases
Targeting Rare, Immunologic Disorders
Harnessing a Natural Mechanism to Silence Disease
Advancing Precision Medicine with Patient Data
Pregnancy and Motherhood for a Woman with SMA
Steps to Improve Data Gathering of Rare Diseases
Rare Patients Learn to Make Their Voices Heard
Empowering Patient Organizations with Real-World Data
A Healthcare Communications Student Gets an Education as a Patient
Expanding the CRISPR Toolkit
Marrying Antibodies to RNA Therapies to Target Previously Inaccessible Tissues and Cells
Expanding Access to Whole Genome Sequencing Across the Globe
A First-in-Class Approach to Treating a Rare and Chronic Liver Disease
A New Approach to Treating a Rare Endocrine Disorder
Detecting Genetic Disease Prior to Birth
Turning Words into Action: Equity, Diversity, and Inclusion in Rare Disease
Chasing Cures
A Map to Navigate A Rare Disease
A Popular Cancer Target Is Eyed for a Rare Immune Disorder
Leveraging a Gene Therapy Approach to Maximize Speed and Minimize Costs
Programing the Body to Make Its Own Medicine with Gene Therapy
Developing in Vivo Gene Editors that Target Liver Diseases
A Find-and-Replace Approach to Fixing the Mutation Underlying Sickle Cell Disease
A Bet on a Unique Set of Vectors
How a Rare Diagnosis Impacts a Family
Transforming the Rare Disease Landscape with Data
Creating a Playbook for Bespoke Gene Therapies
Finding Answers for Undiagnosed Patients with Rare Genetic Diseases
How a Familiar Face Can Lead to a Rare Disease Diagnosis
Bringing the Trial to the Patient
Targeting Rare and Chronic Kidney Diseases
Addressing the Delivery Challenges of Genetic Medicines
Targeting Tissues Throughout the Body with RNA Therapies
Delivering Rare Disease Therapies to Patients in Need
An Advocate's Journey through Diagnosis, Loss, and Hope
Using Model Systems to Find Drugs to Repurpose for Rare Diseases
How a Small and Young Foundation Catalyzed Research into a Rare Disease
Overcoming the Limitations of Conventional Cell Engineering
Experimental Therapy for Rare Endocrine Disorder Offers Hope of Improved Care
Forging Gene Therapy Capacity and a Pipeline at the Same Time
Using A Natural DNA Repair Process to Improve Genetic Medicines
Empowering Rare Disease Patients with their Own Health Records
Targeting CNS Disease with Gene Therapies
A New Therapy Offers A Different Approach to Inhibiting the Complement System
Bringing Aberrant Proteins Back into the Fold
A Cat who Curates Rare Mice
Putting the Patient at the Center of Rare Disease Clinical Trials
Creating a Toolkit to Accelerate the Development of Gene Editing Therapies
PTC Looks to Advanced Therapies
Going to Extreme Lengths
Everything’s up to Date in Kansas City, at Least When It Comes to Genomics
Choosing the Right Viral Vector for a Gene Therapy
A Software Platform to Give Patients the Tools to Build Treatments for Rare Genetic Diseases
Side Effects: The Toll a Rare Disease Can Take on a Family in Pursuit of a Cure
Realizing the Potential of CRISPR Gene Editing
Understanding the Economic Toll of Rare Disease in the United States
The Promise of Gene-Based Therapies for Neurodegenerative Conditions
Moving Beyond Viral Vectors for Gene Therapies
Acquisitions Help Jazz Build Toward Commercial Crescendo
Powerful Gene Editing Approach Offers the Promise of Correcting a Range of Rare Diseases