All episodes

A Child's-Eye-View of Clinical Trials

Using Nanoliposomes to Make Cancer Therapies Safer and More Effective

Accelerating Treatments for Rare Disease through Data Sharing

Treating the Root Cause of Sickle Cell Disease

Targeting a Common Pathway in Genetic Forms of Obesity

After Pruning Orchard, Gaspar Focuses on High Value Opportunities

Writing a New Chapter of Genetic Medicine

How an Ultra-Rare Disease Patient Organization Drove Research to a Treatment

With a New Identity, Travere Thearpeutics Seeks to Find Its True Path

Making Gene Therapies Accessible to Patient with Ultra-Rare Conditions

Using RNA Therapies to Target Inherited Retinal Diseases

Part 4-Gene Therapy: A Researcher’s Perspective

Part 3-Gene Therapy: An Industry Perspective

Part 2-Gene Therapy: A Patient Advocate's Perspective

Part 1-Gene Therapy: A Genetic Counselor’s Perspective

An App to Make Patients the Experts in their Own Conditions

Pursuing a New Target for Autoimmune Diseases

Finding a Rare Friend

Industrializing Individual Therapies for People with Rare Conditions

Rare Disease Advocate Turned Filmmaker Turns Lens on his Son

Inhibiting Signaling that Drives Growth of Rare Malformations

Readying a Rare Disease Community for the Availability of Treatments

Forging a Rare Disease Research Agenda

Delivering Injectable Drugs Orally

What Young Adults with Rare Conditions Need to Know About Health Insurance

Mirum Prepares to Seek Approval for Rare Liver Disease Therapy

Payer Perceptions of Fairness in Pricing Rare Disease Therapies

Improving Narcolepsy Therapy with Drug Delivery Technology

Fighting the Persistent Cough in a Rare and Deadly Lung Disease

Targeting Huntington's Disease with Antisense

Assembling a Pipeline for Rare GI Disorders

Treating Huntington's Disease by Addressing Neuroinflammation

How Patient Advocate-Led Research Can Alter a Rare Disease Landscape

Keeping Clinical Trials Going During a Pandemic

Looking for Commonalities in Rare

Learning to Be Comfortable Being Uncomfortable

An Approach to De-Risk Rare Disease Drug Development

Targeting Epigenetics in Rare Proliferative Bone Marrow Cancers

Chiesi Charges Ahead in Rare Disease

Targeting the Metabolism of Rare Cancers

Unlocking Bryson's Brain

Living Therapies for Chronic Conditions

A One-to-Many Approach to Gene Therapy for Retinal Disease

Targeting an Immune Switch in Rare Diseases

Targeting the Regulatory Genome to Treat Rare Blood Diseases

A Reborn Forma Therapeutics Focuses on Rare Blood Diseases and Cancer

Targeting Genetic Diseases at Their Root Cause

Targeting Hormone Dysregulation in Prader-Willi Syndrome

Rare Disease Advocates Take to the Hill

Targeting a Common Link in Otherwise Different Rare Diseases

Bringing Clinical Trials to Patients

Crowdsourcing Therapeutic Approaches for an Ultra-Rare Disease

Delivering Oligonucleotide Therapies to Any Cell Type

Protalix Looks Beyond Biobetters

Driving N-of-1 Therapies for Ultra-Rare Disease Patients

Cutting the Time and Cost of Drug Discovery with MicroOrgans

Acquisition Combines Two Pipelines for Rare Skin Diseases

Programming Cells to Produce Neuroprotective Factors to Treat ALS

Origin's Rapid Path to Seek Approval for Treatment of Ultra-Rare Metabolic Condition

Developing a Common Language for Rare Diseases

Why Payers Lag Drug Developers and Regulators in Embracing Real-World Evidence

How a Brother's Love Is Forging a Path for Customized Therapies for Rare Disease Patients

Addressing the Barriers to Accessing Experimental Therapies

A Journey to Advocacy

Turning Chronic Illness into a Fashion Statement

Connecting Young Adults with Rare and Chronic Conditions

Helping Caregivers Care for Themselves

A Festival of Moving Pictures

Deep Genomics Boasts an AI First in Drug Discovery

How to Live with a Disability with Your Mojo Intact

Why a Big Pharma Exec Embraced Rare Neurological Diseases

Fighting for Other People's Children

A Doctor's Quest to Cure His Own Rare Disease

Susannah Cahalan and her Month of Madness

The Benefits Support Groups Offer Rare Disease Patients

Targeting the Epigenetic Drivers of Cancer

Tackling Kidney Diseases with Orally-Delivered Enzymes

Giving Voice to Patients through Data

Seeking to Halt and Reverse Fibrotic Diseases

Addressing the Complexities of Rare Disease with Centers of Excellence

Using Cell Therapies to Restore Power in Mitochondrial Diseases

Passage Bio Betting on Penn Connection to Give It an Edge in Developing Gene Therapies

Finding Appropriate Endpoints and Measures for Rare Eye Diseases

Marrying Antibodies and Oligonucleotides to Target Genetic Medicine

What Rare Disease Patients Should Understand about Genetics

Program Seeks to Drive Collaborative Research Model Among Rare Disease Groups

A Gene Hunter Becomes a Drug Hunter Too

The Psychological Toll Rare Diseases Can Have on Healthy Siblings

Blood Test Can Provide Earlier Diagnosis of Autism Spectrum Disorder

Understanding the Role of Natural History Studies in Clinical Trials

How Two Mothers Discovered That a Drug for One Rare Disorder Might Benefit Another

Shortening the Diagnostic Odyssey

Eliminating Barriers to Adoption of Children with Rare Diseases

A Young Rare Disease Advocate Discusses Life with an Undiagnosed Condition

The Benefits of an Open-Source Registry for Rare Diseases

How a Patient Group Helped Drive Drug Development in Rett Syndrome

How Misperceptions Can Create Barriers to Care

Targeting Repeat Expansion Disorders with Next-Gen Antisense Drugs

Getting Misfolding Proteins to Shape Up

A Different Approach to Inhibiting the Complement System