All episodes
Innovative Collaboration Model Drives Treatment for Rare Disease
Girl with Rare Disease Inspires Search for Bone Marrow Donors
A Quest for Tribe: Searching for Others with the Same Ultra-Rare Mutation
Raising Awareness in a Town with a History of a Rare Disease
Helping Rare Disease Patients Navigate the World of Information
Overcoming the Challenges of Rare Disease Drug Development
Moving Rare Disease Therapies from Hope to Reality
Children’s National Launches First-of-its-Kind Rare Disease Center
A Common Link in a Group of Rare Diseases Creates an Opportunity for Repurposing
Sobi Works to Expand Rare Disease Indications for Its Drugs
Why Rare Disease Patients Should Work Together to Address Common Concerns
Solving Medical Mysteries with the Wisdom of Crowds
Embracing Collaborations to Drive Development of Rare Disease Therapies
Determining If a Child's Neurologic or Psychiatric Symptoms are Caused by an Infection
Helping Patients with the Same Undiagnosed Genetic Condition Find Each Other
An Entrepreneur Uses a Silicon Valley Approach to Tackle Rare Disease
Looking for Love with a Rare Disease
Why Genomics May Be in Google's DNA
The Power of Small Grants to Make Big Impacts
Building an International Registry for Rare Diseases
Discovering the Creative Use of Outrage
Diagnosing Rare Diseases with Facial Analysis
Charles River Sees Opportunity in Fostering Rare Disease Collaborations
Abeona Advancing Pipeline of Gene Therapies for Rare Diseases
A Patient Attends "an Academic Conference for Everyone"
Teen Advocate Preaches Message of Kindness
A Controversial Approval for a Duchenne Drug and What's Ahead
Matchmaker Exchange Helps Rare Disease Community Find Me a Find
The Promise of RNAi for Hereditary Angioedema and Other Rare Diseases
Why Natural History Studies Matter to Rare Disease Patients
Harnessing Big Data to Work for Rare Disease Patients
Lessons from Autism on Accelerating Drug Development
How One Rare Disease Group Leverages Relationships with Industry
Ice Bucket Challenge Begins to Yield Results
Why a Common Diabetes Drug May Be Able to Treat a Rare Disease
How to Improve Patient Access to Experimental Drugs
Using Social Media to Find Rare Disease Patients for Clinical Trials
How Rare Disease Patients Weigh Risks and Benefits of a Therapy
Why Homology Medicines Might Be a Game Changer for Gene Therapy
Orchard Hopes to Bear the Fruit of Gene Therapy
Novel Approach to Treat Rare Disease May Lead to Drugs for Common Ones
Brining Whole Genome Sequencing into the Clinic
Bringing Patient-Centricity to Clinical Trials
Determining If a Child's Neurologic or Psychiatric Symptoms are Caused by an Infection
Making Genetic Data Accessible to Researchers
How New Sequencing Technology Is Changing Our Understanding of Rare Diseases
Marathon Readies DMD Drug for FDA
Searching for Genes They May Protect Against Deadly Diseases
Helping Patients with the Same Undiagnosed Genetic Condition Find Each Other
Living with a Disease in Search of a Name
New Means of Regulating Genes May Hold Promise for Rare Disease Patients
Invitae Seeks a Faster, Cheaper Path to Diagnosing Genetic Diseases
Filmmaker Turns Lens on the Rare Disease Community
The Case for Keeping Orphans Where They Can Thrive
Rare Disease Legislation Advancing at National, State Level
The Evolving Relationship Between Patient Advocates and Pharma
Phil Reilly on The Quest to Save Children with Rare Genetic Disorders
Unlocking the Genetic Mysteries of Rare Diseases
A Vision for Making Kansas City a Rare Disease Center of Excellence
A Push in Canada for a National Rare Disease Strategy
Jazzed in Utah for World Rare Disease Day
Group Aims to Accelerate Repurposing of Drugs to Treat Rare Diseases
Gene Therapies Bring New Hope to Rare Disease Patients
Why Rare Disease Research Should Matter to Everyone
Researchers Say Reform of the Orphan Drug Act is Needed
Looking for Love with a Rare Disease
Gene Editing Tool Holds Promise in Rare Disease Treatments
A Cross Country Quest for Marrow Donors
Building Strength by Sharing Stories
North Carolina Seeks Leadership Role in Combating Rare Diseases
The Power of Small Grants to Make Big Impacts
Tearing Down Differences with Film
Educating Docs to Improve Diagnosis and Treatment of Genetic Metabolic Diseases
Providing Young Adult Cancer Patients and Survivors Resources They Need
Gene Therapy Company Born from Mother's Quest to Cure Daughter
Accelerating Rare Disease Research through Collaboration
Cracking the Genetics of Rare Diseases through Crowdsourcing
Teen Advocate to World: "We are More than Our Diseases"
Why the 21st Century Cures Act May Be in Trouble in the Senate
How Lisa Bentley Became a World Class Triathlete with Cystic Fibrosis
Understanding the Rare Disease Caregiver
Addressing Life-Threatening Rare Diseases with Gene Therapy
Living with, Rather Than for, a Rare Disease
An Entrepreneur Uses a Silicon Valley Approach to Tackle Rare Disease
Chronicling the Genomic Revolution
How a Radio Personality Became a Rare Disease Advocate
Why Genomics May Be in Google's DNA
An FDA Perspective on Rare Diseases
Helping Patients Take Control of Their Healthcare
Living Life Beyond Limits
Noah Coughlan Completes His 3,000 Mile Trek for Rare Diseases
How the 21st Century Cures Act Puts Patients at the Table
Using Cell and Gene Therapies to Treat Rare Skin Diseases
Incentives Boost Innovation in Rare Disease Therapies
Tensions Brew Between Pharma and Patient Advocacy Groups
The Attraction of Being a Rare Disease Drug Company
Confronting Genetic Destiny in The Lion's Mouth Opens
Royal Pains Throws Spotlight on Rare Diseases
Making the Case Rare Diseases are Fundamental
Programming the Immune System to Produce Drugs