All episodes
Looking Back in Time to Find Rare Disease Patients Today
Ionis CEO Stanley Crooke Discusses Success as a Platform Technology Company
Orchard Looks toward Harvesting Expanded Gene Therapy Pipeline
Connecting Rare Disease Patients in India and the United States
Regenerative Medicine Moves into the Spotlight
Learning New Tricks from Pathogenic Bacteria to Target the Microbiome
Orphan Drug Accelerator Spin-Out Tackles Rare Endocrine Disorders
Aspa Therapeutics Forges Gene Therapy for Rare Neurological Condition
How Patient-Led Collaborations Are Transforming Rare Disease Drug Discovery and Development
Sobi Expands Its Immunology Franchise with HLH Drug Approval
Learning to Thrive
Late-Stage Trial Underway for Treatment of Rare Obesity Disorder
RegenxBio Bets Its Gene Therapy Technology Provides Competitive Edge
The Potential for Digital Health Technologies to Transform Clinical Trials
Matching Volunteers to Rare Disease Organizations in Need
How One Rare Disease Organization Used Technology to Provide Greater Patient Insight to FDA
The Potentially Deadly Consequences of an Oversight in Newborn Screening Tests
Advancing a New Approach to Sickle Cell Disease
Conquering Life with a Rare Disease
Why Rare Disease Drug Developers Need Regular Interaction with the FDA
Making the Case for an FDA Rare Disease Center of Excellence
An Elite Runner Makes a Cross-Canada Trek for His Son and Rare Disease
How Rare Disease Advocates Can Get The Most out of Collaborations
Establishing Clinical Standards for a Rare Disease
Teen Rare Disease Advocate Fights the Condition That Took His Brother's Life
Addressing the Emotional Toll of Rare Diseases
Forging a Research Agenda for a Rare Disease
Living with an Uncertain Rare Diagnosis
A Rare Disease Advocate Teaches Doctors to See and Speak Differently
The Caregiver Who Cried in the Shower
Curating Patient Data to Accelerate Rare Disease Drug Development
Forging a Research Agenda for a Rare Disease
Making the Journey from All-American Athlete to Rare Disease Advocate
How Destiny Lamonte Learned to Advocate for Herself and Others
Where the NIH Does Its Banking
Making Sense of Nonsense Mutations
Rewriting Faulty RNA
Experimental Drug for "Elephant Man" Disease May Benefit Certain Cancers Too
Programming Stem Cells to Protect Neurons in ALS and other Neurodegenerative Conditions
Why a Topical, Off-the-Shelf, Gene Therapy May Hold Promise for a Rare Skin Condition
An Ironman Champion with Cystic Fibrosis Discusses her Changing Relationship with Her Disease
Why an Experimental Fragile X Drug May Treat a Range of Neurodegenerative Diseases
The Case for a Daily Self-Injection to Treat Hemophilia
Addressing the Challenges of R&D for Rare Cancer Therapies
Learning to Communicate and Collaborate as a Patient Advocate
Seeking Answers for Undiagnosed Patients
When "Undiagnosed" Is Your Diagnosis
The Decision to Get Tested for Huntington's Disease and How It Changed Seth Rotberg's Life
Nonprofit Sees Need in Developing Gene Therapies for Inherited Eye Disesases
Casting a Wider Net for Genetic Diseases
Corbus Seeks to Quiet the Immune System in Chronic Conditions
Addressing the Challenges of Rare Disease Drug Development
Giving Voice to Patients at the FDA
Finding a Rare Friend
Why a Sports Physical May Trigger a Doctor to Think About a Rare Diagnosis
UPenn's Orphan Disease Center Takes a Rare Approach
How Patients Can Accelerate Translational Research in Rare Disease
Notre Dame Center Leverages Commonalities of Rare and Neglected Diseases
GalXC Quest: Dicerna Bets on Its Unqiue Approach to RNAi
Pharnext Takes a Unique Twist on Repurposing Drugs for Rare Diseases
uniQure Seeks to Find Elusive Market Success with Hemophilia B Gene Therapy
Savara Advances Pipeline of Inhaled Therapeutics for Rare Lung Diseases
Why the Microbiome May Provide a Novel Approach to Treating Certain Rare Diseases
FDA Outlines a Collaborative Approach for Pediatric Rare Disease Trials
Why Patient-Centred Outcomes Are Critical to Rare Disease Drug Trials
What the FDA's New Framework Means for Regenerative Therapies
Measuring the Value of Therapies for Ultra-Rare Diseases
Lessons from Pitching Venture Capitalists
Health Canada Scraps Plans for Orphan Drug Framework
New Investment Boosts Rare Disease Drug Accelerator
Understanding Rare Disease Caregiver Needs
Deriving Rare Disease Therapies from Human Plasma
Learning to Live with Purpose
A Mother's Race to Find a Treatment for Her Daughter's Ultra-Rare Condition
Screening for Rare Diseases
A Look at the Changing World of Work for People with a Chronic Illness
Aligning Interests in Rare Disease Partnerships
A Veteran Venture Capitalist Discusses Investing in Rare Disease Drug Development
A Patient Group Crafts Guidelines for Working with Pharma
Bringing Innovation to Business Models for Rare Disease Drug Development
Rethinking Accessibility and Dependency
How Simon Wheatcroft, Blinded by a Rare Disease, Became an Ultramarathoner
Spark Hoping Its Luxturna Will Become First FDA-Approved Gene Therapy
What Rare Patient-Investors Can Learn From Venture Capitalists
Novel Effort Delivers Failed DMD Drug to Former Clinical Trial Participants
Centogene Seeks to Deliver Diagnoses to Rare Disease Patients Early
Albireo Advances Pipeline for Rare Liver and Gastrointestinal Diseases
aTyr Pharma Seeks to Modulate Activity in FSHD and Other Rare Disease
Summit Pursues A Different Approach to Treating Duchenne
Delivering Enzyme Replacement Therapies Across the Blood-Brain Barrier
Sangamo Advances Gene Editing Therapies for Multiple Rare Diseases into the Clinic
Navigating the Challenges of Rare Disease Drug Development
Accelerating Rare Disease Drug Discovery
Experimental Rare Disease Therapy Wins New FDA Reg Med Designation
How Rare Disease Patients Can Get their Voices Heard in Drug Discovery and Development
One Patient's Difficult Road to a Rare Diagnosis
How Researchers Diagnosed Four Patients with a Never-Before-Identified Rare Disease in a Day
Stem Cell Gene Therapy Restores Immune System in Kids with SCID
Rare Disease Advocates Push for Incentives to Drugmakers Pulled from Cures Act
Non-Profit Drug Company Seeks to Advance Rare Disease Therapies