RARECast
By RARECast
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Episodes: 595
Description
RARECast is a Global Genes podcast hosted by award-winning journalist Daniel Levine. It focuses on the intersection of rare disease with business, science, and policy.
| Episode | Date |
|---|---|
|
Raising Awareness of Clinical Trials
|
May 14, 2026 |
|
Using AI and Longitudinal Data to Transform Rare Disease Care
|
May 07, 2026 |
|
Cutting through the Diagnostic Maze for Rare Diseases
|
Apr 30, 2026 |
|
How Parents Took Development of a Gene Therapy into Their Own Hands
|
Apr 23, 2026 |
|
Matching the Right Therapy to the Right Child with a Rare Cancer
|
Apr 16, 2026 |
|
From Treating Symptoms to Addressing Causes in Rare Autoimmune Diseases
|
Apr 09, 2026 |
|
A Mother’s Journey to Rewrite a Neurodevelopmental Disorder
|
Apr 02, 2026 |
|
How a Foundation Built Its Own Drug Program for an Ultra-Rare Disease
|
Mar 26, 2026 |
|
From Bloodletting to Breakthroughs in PV
|
Mar 19, 2026 |
|
When Endpoints Miss the Point
|
Mar 12, 2026 |
|
Reopening the Developmental Window in Rett Syndrome with a Gene Therapy
|
Mar 04, 2026 |
|
A Data Strategy to Capitalize on a Multi Trillion Dollar Opportunity
|
Feb 26, 2026 |
|
When a Gene Echoes Through a Family
|
Feb 19, 2026 |
|
Changing the Autoimmune Disease Playbook with RNA-Engineered CAR T Cells
|
Feb 12, 2026 |
|
Rewriting Rare Disease R&D with Foundation Models
|
Feb 05, 2026 |
|
Resetting Aberrant Tregs Epigenetically to Treat Autoimmune Diseases
|
Jan 29, 2026 |
|
Finding a Ready Treatment for a Newly Discovered, Ultra-Rare Disease
|
Jan 22, 2026 |
|
One Rare Mother’s Quest to Rewrite Her Son’s Future with a Gene Therapy
|
Jan 15, 2026 |
|
Transforming TSC Epilepsy with a Precision Therapy
|
Jan 08, 2026 |
|
Bridging the Valley for Rare Disease Drug Development
|
Jan 01, 2026 |
|
Turning a Rare Cancer into a Call to Action
|
Dec 24, 2025 |
|
Developing a New Approach to Cystic Fibrosis to Improve Outcomes
|
Dec 18, 2025 |
|
Finding Strength in Weakness
|
Dec 11, 2025 |
|
Developing Targeted Therapies for a Rare Autoimmune Disease
|
Dec 04, 2025 |
|
A Venture Fund that Leverages Patient Experts to Target Autoimmune Diseases
|
Nov 26, 2025 |
|
Navigating Your Child’s Rare Disease
|
Nov 20, 2025 |
|
Changing a Treatment Landscape by De-Risking Drug Development
|
Nov 13, 2025 |
|
Transforming Real-World Patient Data into Breakthroughs
|
Nov 06, 2025 |
|
Taking Aim at Huntington’s Disease and Other Repeat Expansion Disorders
|
Oct 30, 2025 |
|
Learning to Be a Caregiver and Advocate
|
Oct 23, 2025 |
|
Improving Rare Disease Care through Partnerships
|
Oct 16, 2025 |
|
A Call on the FDA for Timely Reviews of Life-Saving Therapies for Rare Diseases
|
Oct 09, 2025 |
|
The Need to Look Beyond Seizures When Treating DEEs
|
Oct 02, 2025 |
|
Taking On Big Competitors with an Oral Therapy to Treat Achondroplasia
|
Sep 25, 2025 |
|
Going Viral to Fight Brain Cancer
|
Sep 18, 2025 |
|
Changing the Playbook on Childhood Cancers
|
Sep 11, 2025 |
|
Sometimes the Hoofbeats Do Belong to Zebras
|
Sep 04, 2025 |
|
Breathing Easier with a Breakthrough Immunotherapy
|
Aug 28, 2025 |
|
Using AI Open Innovation to Tackle ALS
|
Aug 21, 2025 |
|
Recovering What’s Lost in Translation
|
Aug 14, 2025 |
|
Targeting Hunger Rather than Appetite in Prader-Willi Syndrome
|
Aug 07, 2025 |
|
A Rare-to-Common Business Model
|
Jul 31, 2025 |
|
Leading a Rare Youth Movement
|
Jul 24, 2025 |
|
Developing Gene Therapies that Work Across Mutations
|
Jul 17, 2025 |
|
Eyeing A Challenge to a Blockbuster
|
Jul 10, 2025 |
|
Like Mother, Like Child
|
Jul 03, 2025 |
|
Transforming from Grandmother to Mother to Advocate
|
Jun 26, 2025 |
|
Overcoming a Delivery Challenge of Retinal Gene Therapies
|
Jun 19, 2025 |
|
A Gene Editing First Augurs an Era of Bespoke Therapies
|
Jun 12, 2025 |
|
How One Patient Organization Drives Drug Development
|
Jun 05, 2025 |
|
Using CRISPR to Modulate Gene Expression
|
May 29, 2025 |
|
Satisfying the Hunger for a Prader-Willi Therapy
|
May 22, 2025 |
|
How an Academic Medical Center Helped Change the Landscape for a Rare Disease
|
May 15, 2025 |
|
Improving Outcomes for People with a Set of Rare Cancers
|
May 08, 2025 |
|
An Effort to Build a Better DMD Gene Therapy
|
May 02, 2025 |
|
A Once-Failed Pain Therapy Shows Potential in a Neurodevelopmental Disorder
|
Apr 24, 2025 |
|
Advancing a Cutting-Edge Therapy for a Rare, Childhood Cancer
|
Apr 17, 2025 |
|
Learning to Take His Vitamins
|
Apr 10, 2025 |
|
The First Treatment for a Rare Neurodegenerative Condition Awaits FDA Approval
|
Apr 03, 2025 |
|
Engineering Skin Bacteria to Be Live Biotherapeutics
|
Mar 27, 2025 |
|
Seeing the Gene and Cell Therapy Translational Divide as an Opportunity
|
Mar 20, 2025 |
|
My Mother, Myself, and ALS
|
Mar 13, 2025 |
|
From Immovable Object to Advocacy Force
|
Mar 06, 2025 |
|
A Rapid and Scalable Approach for Screening Personalized ASOs
|
Feb 27, 2025 |
|
Addressing the Disease Mechanism of a Rare Kidney Disease
|
Feb 20, 2025 |
|
The Mother of a Son with SCD, Applies Direct Experience to Her Clinical Trials Work
|
Feb 13, 2025 |
|
A First for Rett Syndrome with More in the Pipeline
|
Feb 06, 2025 |
|
A Heart Attack that Led to a Rare Disease Diagnosis
|
Jan 30, 2025 |
|
Targeting Rare Liver Diseases with Gene Editing Therapies
|
Jan 23, 2025 |
|
Using AI to De-Risk Rare Disease Drug Development
|
Jan 16, 2025 |
|
Addressing a Growing Demand for Plasma-Derived Therapies
|
Jan 09, 2025 |
|
Empowering Patients with Data to Drive Drug Development
|
Jan 02, 2025 |
|
How Advocates Are Advancing a Treatment for an Ultra-Rare Disease
|
Dec 26, 2024 |
|
Engineering B Cells to Produce Therapeutic Proteins
|
Dec 19, 2024 |
|
A Pipeline Win as BridgeBio Learns to Balance Theory with Practice
|
Dec 12, 2024 |
|
Considering What It Will Take for Payers to Embrace Advances in Sequencing
|
Dec 05, 2024 |
|
Finding Strength
|
Nov 27, 2024 |
|
A Non-Profit Works to Advance a DMD Gene Therapy
|
Nov 21, 2024 |
|
Accepting What We Can’t Understand
|
Nov 14, 2024 |
|
A Gene Therapy Company Born from a Father’s Efforts to Save his Daughter
|
Nov 07, 2024 |
|
Lowering Barriers to Clinical Trial Participation with Do-It-Yourself Blood Sample Collection
|
Oct 31, 2024 |
|
Scaling N-of-1 Therapies to Viability
|
Oct 24, 2024 |
|
Building Gene Therapies that Address Gain and Loss of Function Simultaneously
|
Oct 17, 2024 |
|
How One Woman Climbed from Patient to Advocate
|
Oct 10, 2024 |
|
Shortening the Diagnostic Odyssey
|
Oct 03, 2024 |
|
Making Exomes More Revealing
|
Sep 26, 2024 |
|
How One Patient Organization Catalyzed Drug Development
|
Sep 19, 2024 |
|
Enabling On-Demand Therapies
|
Sep 16, 2024 |
|
After Driving an N-of-1 Therapy for Her Son, an Advocate Turns to Helping Others
|
Sep 05, 2024 |
|
Putting Rare Disease Organization in the Business of Drug Development
|
Aug 29, 2024 |
|
Understanding the Many Questions Gene Therapies Raise
|
Aug 22, 2024 |
|
A Test to Identify People Likely to Develop ALS
|
Aug 15, 2024 |
|
How an Ultra-Rare Disease Patient Foundation Advanced a Gene Therapy
|
Aug 08, 2024 |
|
Making the $10 Genome a Reality
|
Aug 01, 2024 |
|
A No-Nonsense Effort to Develop a Therapy that Works Across Genetic Diseases
|
Jul 25, 2024 |
|
Mapping a Perilous Journey with Humor
|
Jul 18, 2024 |
|
Restoring Vision in Inherited Retinal Disease
|
Jul 11, 2024 |
|
Bringing Genome Sequencing to Rural Populations
|
Jul 04, 2024 |
|
Addressing a Blind Spot in Treatments for the Cornea
|
Jun 27, 2024 |
|
Taking a Different Approach to Rare Epilepsies
|
Jun 20, 2024 |
|
A Rare Disease Drug Developer Tries to Earn Its Stripes
|
Jun 13, 2024 |
|
Using Plasma to Treat Rare Diseases
|
Jun 06, 2024 |
|
Differentiating Gene Therapies through Regulatory Elements
|
May 30, 2024 |
|
How One Patient Organization Leverages Research Investments
|
May 23, 2024 |
|
Charging into the Storm
|
May 16, 2024 |
|
A Gene Therapy Developer that Embraces Different Models for Reaching Patients
|
May 09, 2024 |
|
Using Directed Evolution to Develop New Vectors for Genetic Medicines
|
May 02, 2024 |
|
Addressing a Shortage of Genetic Counselors with AI
|
Apr 25, 2024 |
|
A Small Molecule Therapy to Regenerate Muscle in People with DMD
|
Apr 18, 2024 |
|
Forging a Faster Path for Gene Therapies
|
Apr 11, 2024 |
|
Determining the Value of Rare Disease Therapies
|
Apr 04, 2024 |
|
Buying and Building a Gene Therapy Presence
|
Mar 28, 2024 |
|
A Clinical Trial Failure Derails a Promising Technology
|
Mar 21, 2024 |
|
Helping People with Undiagnosed Rare Diseases Find Answers
|
Mar 14, 2024 |
|
Speeding and Scaling the Development of Genome Editing Therapies
|
Mar 07, 2024 |
|
Getting a Next-Generation Genome Editing Therapy for Sickle Cell Disease Back on Track
|
Feb 29, 2024 |
|
Trying to Break a Leg in a Wheelchair
|
Feb 22, 2024 |
|
A Drug Developer that Makes Pediatric Cancers a Priority
|
Feb 15, 2024 |
|
Harnessing the Body’s Natural RNA Machinery to Treat Diseases
|
Feb 08, 2024 |
|
Targeting the Leading Cause of Death in Friedreich’s Ataxia with a Gene Therapy
|
Feb 01, 2024 |
|
How Inhaled mRNA May Help Rare Disease Patients Breathe Easier
|
Jan 25, 2024 |
|
Changing What’s Possible with Cell and Gene Therapies
|
Jan 18, 2024 |
|
Why Flying with a Wheelchair Is a Civil Rights Issue
|
Jan 11, 2024 |
|
Mapping Spheres of Influence
|
Jan 04, 2024 |
|
Bringing Precision to the Treatment of Rare Cancers
|
Dec 28, 2023 |
|
The Challenges of Developing a Therapy for Pregnant Women with A Rare Condition
|
Dec 21, 2023 |
|
How One Drug Developer Is Addressing Health Inequities
|
Dec 14, 2023 |
|
Accelerating the Development of Genetic Medicines with AI
|
Dec 07, 2023 |
|
Leveraging AI to Address the Neuropsychiatric Conditions Associated with a Rare Disease
|
Nov 30, 2023 |
|
In Pursuit of a Treatment for His Son, A Lawyer Becomes a Biotech Executive
|
Nov 22, 2023 |
|
In Push for Treatment, a Patient Organization Becomes a Trial Sponsor
|
Nov 16, 2023 |
|
Delayed Diagnosis of Rare Diseases Takes Staggering Economic Toll
|
Nov 09, 2023 |
|
A Cell Therapy to Control Heart-Damaging Inflammation in Duchenne
|
Nov 02, 2023 |
|
Stimulating the Growth of Muscle Mass in People with Neuromuscular Diseases
|
Oct 26, 2023 |
|
Turning Grief into Action
|
Oct 19, 2023 |
|
A Plea to the FDA to Give an Ultra-Rare Disease Drug a Fair Hearing
|
Oct 12, 2023 |
|
How the Woolly Mammoth Beat a Path to Better Gene Therapies
|
Oct 05, 2023 |
|
A Genetic Counselor’s Journey into Patient Advocacy
|
Sep 28, 2023 |
|
A Company in the Fold of Rare Disease
|
Sep 21, 2023 |
|
Disabling Antibodies that Drive Rare, Immunological Conditions
|
Sep 14, 2023 |
|
Keeping the Sentinels of the Brain Vigilant
|
Sep 07, 2023 |
|
Considering the Unfinished Work in Cystic Fibrosis
|
Aug 31, 2023 |
|
How to Advocate for Yourself as You Battle Cancer
|
Aug 24, 2023 |
|
Targeting Neuroinflammation to Treat ALS
|
Aug 17, 2023 |
|
Leveraging Community Partnerships to Address a Rare Disease Behind a Medical Mystery in New Mexico
|
Aug 10, 2023 |
|
Accelerating Gene Editing Therapies for Rare, Neurological Conditions
|
Aug 03, 2023 |
|
Inhibiting Electrical Activity in Rare, Seizure Disorders
|
Jul 27, 2023 |
|
Improving the Function and Usability of Clothing for People with Disabilities
|
Jul 20, 2023 |
|
Treating Cardiopulmonary Disease with Inhaled Targeted Therapies
|
Jul 13, 2023 |
|
A Patient-Driven Registry Focused on Health-Related Quality of Life Data
|
Jun 29, 2023 |
|
An Ally for the Undiagnosed Emerges
|
Jun 29, 2023 |
|
Help for Rare Disease Patients That’s Just a Click Away
|
Jun 22, 2023 |
|
Diagnosing Autism with a Single Strand of Hair
|
Jun 15, 2023 |
|
Bringing Regenerative Medicine to a Rare Bone Condition in Children
|
Jun 08, 2023 |
|
Bringing Precision to CRISPR-Based Genome Editing
|
Jun 01, 2023 |
|
Capturing Patients’ Experience in Their Daily Lives
|
May 25, 2023 |
|
From One to Many
|
May 18, 2023 |
|
A Nasal Spray to Curb Excessive Eating in Prader-Willi
|
May 11, 2023 |
|
How One Foundation Is Laying the Groundwork to Advance Treatments for an Ultra-Rare Disease
|
May 04, 2023 |
|
Forging a New Path for Medicine
|
Apr 27, 2023 |
|
Restoring Hearing through Gene Therapies
|
Apr 20, 2023 |
|
Creating a Sustainable Reimbursement Model for Ultra-Rare Therapies
|
Apr 13, 2023 |
|
Lumos Hopes Its Oral Alternative to HGH Will Fuel Its Growth
|
Apr 05, 2023 |
|
Advancing a Gene Therapy for a Rare and Fatal CNS Disorder
|
Mar 30, 2023 |
|
Addressing the Barriers to Patient Participation in Clinical Trials
|
Mar 23, 2023 |
|
A Venture Philanthropist Makes the Case for Advocates Taking Equity
|
Mar 16, 2023 |
|
Seeking a Sustainable Business Developing on N-of-1 Therapies
|
Mar 09, 2023 |
|
How One Drugmaker Reaches out to Communities at Greater Risk for Rare Heart Condition
|
Mar 03, 2023 |
|
A Next-Generation RNA Therapy Targets Telomere Disorders
|
Mar 03, 2023 |
|
BridgeBio Advances Therapy for Limb-Girdle Muscular Dystrophy that Started with Two Patient Families
|
Feb 24, 2023 |
|
Translating Urgency into Science
|
Feb 16, 2023 |
|
Empowering Ultra-Rare Disease Patients to Pursue the Discovery of Treatments
|
Feb 10, 2023 |
|
Why a Topical Cannabidiol Gel May Help Treat the Behavioral Symptoms of Fragile X
|
Feb 02, 2023 |
|
An Ultra-Rare Disease Drug Developer Tries to Navigate Regulatory Uncertainty
|
Jan 26, 2023 |
|
Powering Cells in People with Rare Mitochondrial Diseases
|
Jan 19, 2023 |
|
Powering Weakened and Stressed Cells in ALS to Function Better with Nanocrystal Therapy
|
Jan 12, 2023 |
|
Developing a New Class of Therapies Based on a Natural Cargo Carrier
|
Jan 05, 2023 |
|
Reaching Beyond the Limits of Enzyme Replacement Therapies with Gene Therapies
|
Dec 29, 2022 |
|
One Woman’s Journey as a Caregiver to a Husband with Frontotemporal Dementia
|
Dec 22, 2022 |
|
Treating Rare Endocrine Disorders with Therapeutic Peptides
|
Dec 15, 2022 |
|
Tackling the Pricing Challenges for Advanced Therapies for Rare Diseases
|
Dec 08, 2022 |
|
Addressing the Current Limitations of AAV Gene Therapies
|
Dec 01, 2022 |
|
Examining the Legislative Landscape for Rare Disease Drug Development
|
Nov 24, 2022 |
|
How a Drug Setback Became a Patient Community’s Gain
|
Nov 18, 2022 |
|
Helping Regulators and Drug Developers Understand the Challenges of Living with Fabry Disease
|
Nov 10, 2022 |
|
Addressing Racial Disparities in a Rare Blood Cancer
|
Nov 03, 2022 |
|
How New Sequencing Technology Promises to Alter the Diagnostic Odyssey
|
Oct 27, 2022 |
|
Leveraging Technology to Empower Patients and Decentralize Clinical Trials
|
Oct 20, 2022 |
|
Equipping The Next-Generation Rare Disease Patient Advocate
|
Oct 13, 2022 |
|
How a Family Raced from Diagnosis to Experimental Gene Therapy in Three Years
|
Oct 06, 2022 |
|
How One Advocate Went from Losing His Hearing to Being Heard
|
Sep 29, 2022 |
|
Building a Pipeline of Therapies to Treat Rare Mineralization Disorders
|
Sep 22, 2022 |
|
Advancing an Oral Alternative to Infused and Injected Therapies for HAE
|
Sep 15, 2022 |
|
Embracing the Promise of Patient-Centered Biotechnology
|
Sep 08, 2022 |
|
Learning to Live with Loss
|
Sep 01, 2022 |
|
Using Cryptocurrency to Tap into the Wisdom of Crowds
|
Aug 25, 2022 |
|
A Therapy for a Rare Neurodegenerative Disease Moves Towards Regulatory Review
|
Aug 18, 2022 |
|
Transforming the Treatment of Neuromuscular Diseases with Next-Gen Oligonucleotides
|
Aug 11, 2022 |
|
Teaching the Immune System to Let Medicines Do Their Job
|
Aug 04, 2022 |
|
Designing Clinical Trials with the Patient in Mind
|
Jul 28, 2022 |
|
Keeping Clinical Trials Running Smoothly
|
Jul 21, 2022 |
|
A Vision for Patient-Centric Gene Therapy Development
|
Jul 14, 2022 |
|
Using Genetic Testing to Address Disparities in Care for Kidney Disease
|
Jul 07, 2022 |
|
Why Reforms to the Accelerated Approval Pathway Threaten Rare Disease Drug Development
|
Jun 30, 2022 |
|
A Longstanding Academic-Nonprofit Collaboration Gives Rise to an ALS Drug Company
|
Jun 16, 2022 |
|
Targeting Regulatory RNA to Upregulate Gene Expression to Treat Rare Diseases
|
Jun 09, 2022 |
|
Developing a New Approach to Treat Rare, Autoimmune Conditions
|
Jun 02, 2022 |
|
Bridging the Gap Between Basic and Commercial Research for Rare Disease
|
May 26, 2022 |
|
Stoking Functional Copies of Genes to Compensate for Mutated Ones
|
May 19, 2022 |
|
Targeting ALS with Synergistic Combinations of Therapies
|
May 13, 2022 |
|
How a Polish Rare Disease Organizations Is Helping Its Ukrainian Neighbors
|
May 05, 2022 |
|
Powering a New Era of Genetic Medicine
|
Apr 28, 2022 |
|
Searching for a Diagnosis
|
Apr 21, 2022 |
|
A Novel Way to Deliver Rare Disease Therapies
|
Apr 14, 2022 |
|
Bringing Diversity, Equity, and Inclusion to the Books We Read
|
Apr 07, 2022 |
|
From Mila to Millions: Scaling N of 1 Therapies
|
Mar 31, 2022 |
|
Genetically Modifying Patients’ Skin Cells to Treat Rare Diseases
|
Mar 24, 2022 |
|
Targeting Rare, Immunologic Disorders
|
Mar 18, 2022 |
|
Harnessing a Natural Mechanism to Silence Disease
|
Mar 10, 2022 |
|
Advancing Precision Medicine with Patient Data
|
Mar 03, 2022 |
|
Pregnancy and Motherhood for a Woman with SMA
|
Feb 24, 2022 |
|
Steps to Improve Data Gathering of Rare Diseases
|
Feb 17, 2022 |
|
Rare Patients Learn to Make Their Voices Heard
|
Feb 10, 2022 |
|
Empowering Patient Organizations with Real-World Data
|
Feb 04, 2022 |
|
A Healthcare Communications Student Gets an Education as a Patient
|
Jan 27, 2022 |
|
Expanding the CRISPR Toolkit
|
Jan 21, 2022 |
|
Marrying Antibodies to RNA Therapies to Target Previously Inaccessible Tissues and Cells
|
Jan 13, 2022 |
|
Expanding Access to Whole Genome Sequencing Across the Globe
|
Jan 06, 2022 |
|
A First-in-Class Approach to Treating a Rare and Chronic Liver Disease
|
Dec 31, 2021 |
|
A New Approach to Treating a Rare Endocrine Disorder
|
Dec 24, 2021 |
|
Detecting Genetic Disease Prior to Birth
|
Dec 16, 2021 |
|
Turning Words into Action: Equity, Diversity, and Inclusion in Rare Disease
|
Dec 10, 2021 |
|
Chasing Cures
|
Dec 03, 2021 |
|
A Map to Navigate A Rare Disease
|
Nov 24, 2021 |
|
A Popular Cancer Target Is Eyed for a Rare Immune Disorder
|
Nov 19, 2021 |
|
Leveraging a Gene Therapy Approach to Maximize Speed and Minimize Costs
|
Nov 12, 2021 |
|
Programing the Body to Make Its Own Medicine with Gene Therapy
|
Nov 04, 2021 |
|
Developing in Vivo Gene Editors that Target Liver Diseases
|
Oct 28, 2021 |
|
A Find-and-Replace Approach to Fixing the Mutation Underlying Sickle Cell Disease
|
Oct 21, 2021 |
|
A Bet on a Unique Set of Vectors
|
Oct 15, 2021 |
|
How a Rare Diagnosis Impacts a Family
|
Oct 07, 2021 |
|
Transforming the Rare Disease Landscape with Data
|
Oct 01, 2021 |
|
Creating a Playbook for Bespoke Gene Therapies
|
Sep 24, 2021 |
|
Finding Answers for Undiagnosed Patients with Rare Genetic Diseases
|
Sep 16, 2021 |
|
How a Familiar Face Can Lead to a Rare Disease Diagnosis
|
Sep 10, 2021 |
|
Bringing the Trial to the Patient
|
Sep 02, 2021 |
|
Targeting Rare and Chronic Kidney Diseases
|
Aug 26, 2021 |
|
Addressing the Delivery Challenges of Genetic Medicines
|
Aug 19, 2021 |
|
Targeting Tissues Throughout the Body with RNA Therapies
|
Aug 12, 2021 |
|
Delivering Rare Disease Therapies to Patients in Need
|
Aug 05, 2021 |
|
An Advocate's Journey through Diagnosis, Loss, and Hope
|
Jul 29, 2021 |
|
Using Model Systems to Find Drugs to Repurpose for Rare Diseases
|
Jul 22, 2021 |
|
How a Small and Young Foundation Catalyzed Research into a Rare Disease
|
Jul 16, 2021 |
|
Overcoming the Limitations of Conventional Cell Engineering
|
Jul 08, 2021 |
|
Experimental Therapy for Rare Endocrine Disorder Offers Hope of Improved Care
|
Jul 02, 2021 |
|
Forging Gene Therapy Capacity and a Pipeline at the Same Time
|
Jun 24, 2021 |
|
Using A Natural DNA Repair Process to Improve Genetic Medicines
|
Jun 18, 2021 |
|
Empowering Rare Disease Patients with their Own Health Records
|
Jun 10, 2021 |
|
Targeting CNS Disease with Gene Therapies
|
Jun 03, 2021 |
|
A New Therapy Offers A Different Approach to Inhibiting the Complement System
|
May 28, 2021 |
|
Bringing Aberrant Proteins Back into the Fold
|
May 20, 2021 |
|
A Cat who Curates Rare Mice
|
May 13, 2021 |
|
Putting the Patient at the Center of Rare Disease Clinical Trials
|
May 06, 2021 |
|
Creating a Toolkit to Accelerate the Development of Gene Editing Therapies
|
Apr 29, 2021 |
|
PTC Looks to Advanced Therapies
|
Apr 22, 2021 |
|
Going to Extreme Lengths
|
Apr 15, 2021 |
|
Everything’s up to Date in Kansas City, at Least When It Comes to Genomics
|
Apr 08, 2021 |
|
Choosing the Right Viral Vector for a Gene Therapy
|
Apr 01, 2021 |
|
A Software Platform to Give Patients the Tools to Build Treatments for Rare Genetic Diseases
|
Mar 25, 2021 |
|
Side Effects: The Toll a Rare Disease Can Take on a Family in Pursuit of a Cure
|
Mar 19, 2021 |
|
Realizing the Potential of CRISPR Gene Editing
|
Mar 12, 2021 |
|
Understanding the Economic Toll of Rare Disease in the United States
|
Mar 04, 2021 |
|
The Promise of Gene-Based Therapies for Neurodegenerative Conditions
|
Feb 25, 2021 |
|
Moving Beyond Viral Vectors for Gene Therapies
|
Feb 19, 2021 |
|
Acquisitions Help Jazz Build Toward Commercial Crescendo
|
Feb 12, 2021 |
|
Powerful Gene Editing Approach Offers the Promise of Correcting a Range of Rare Diseases
|
Feb 05, 2021 |
|
A Child's-Eye-View of Clinical Trials
|
Jan 29, 2021 |
|
Using Nanoliposomes to Make Cancer Therapies Safer and More Effective
|
Jan 21, 2021 |
|
Accelerating Treatments for Rare Disease through Data Sharing
|
Jan 14, 2021 |
|
Treating the Root Cause of Sickle Cell Disease
|
Jan 08, 2021 |
|
Targeting a Common Pathway in Genetic Forms of Obesity
|
Dec 30, 2020 |
|
After Pruning Orchard, Gaspar Focuses on High Value Opportunities
|
Dec 23, 2020 |
|
Writing a New Chapter of Genetic Medicine
|
Dec 17, 2020 |
|
How an Ultra-Rare Disease Patient Organization Drove Research to a Treatment
|
Dec 10, 2020 |
|
With a New Identity, Travere Thearpeutics Seeks to Find Its True Path
|
Dec 04, 2020 |
|
Making Gene Therapies Accessible to Patient with Ultra-Rare Conditions
|
Nov 26, 2020 |
|
Using RNA Therapies to Target Inherited Retinal Diseases
|
Nov 20, 2020 |
|
Part 4-Gene Therapy: A Researcher’s Perspective
|
Nov 13, 2020 |
|
Part 3-Gene Therapy: An Industry Perspective
|
Nov 06, 2020 |
|
Part 2-Gene Therapy: A Patient Advocate's Perspective
|
Oct 29, 2020 |
|
Part 1-Gene Therapy: A Genetic Counselor’s Perspective
|
Oct 23, 2020 |
|
An App to Make Patients the Experts in their Own Conditions
|
Oct 15, 2020 |
|
Pursuing a New Target for Autoimmune Diseases
|
Oct 08, 2020 |
|
Finding a Rare Friend
|
Oct 02, 2020 |
|
Industrializing Individual Therapies for People with Rare Conditions
|
Sep 25, 2020 |
|
Rare Disease Advocate Turned Filmmaker Turns Lens on his Son
|
Sep 17, 2020 |
|
Inhibiting Signaling that Drives Growth of Rare Malformations
|
Sep 10, 2020 |
|
Readying a Rare Disease Community for the Availability of Treatments
|
Sep 03, 2020 |
|
Forging a Rare Disease Research Agenda
|
Aug 27, 2020 |
|
Delivering Injectable Drugs Orally
|
Aug 20, 2020 |
|
What Young Adults with Rare Conditions Need to Know About Health Insurance
|
Aug 14, 2020 |
|
Mirum Prepares to Seek Approval for Rare Liver Disease Therapy
|
Aug 07, 2020 |
|
Payer Perceptions of Fairness in Pricing Rare Disease Therapies
|
Aug 04, 2020 |
|
Improving Narcolepsy Therapy with Drug Delivery Technology
|
Jul 23, 2020 |
|
Fighting the Persistent Cough in a Rare and Deadly Lung Disease
|
Jul 15, 2020 |
|
Targeting Huntington's Disease with Antisense
|
Jul 08, 2020 |
|
Assembling a Pipeline for Rare GI Disorders
|
Jul 01, 2020 |
|
Treating Huntington's Disease by Addressing Neuroinflammation
|
Jun 24, 2020 |
|
How Patient Advocate-Led Research Can Alter a Rare Disease Landscape
|
Jun 17, 2020 |
|
Keeping Clinical Trials Going During a Pandemic
|
Jun 10, 2020 |
|
Looking for Commonalities in Rare
|
Jun 03, 2020 |
|
Learning to Be Comfortable Being Uncomfortable
|
May 27, 2020 |
|
An Approach to De-Risk Rare Disease Drug Development
|
May 20, 2020 |
|
Targeting Epigenetics in Rare Proliferative Bone Marrow Cancers
|
May 13, 2020 |
|
Chiesi Charges Ahead in Rare Disease
|
May 06, 2020 |
|
Targeting the Metabolism of Rare Cancers
|
Apr 29, 2020 |
|
Unlocking Bryson's Brain
|
Apr 22, 2020 |
|
Living Therapies for Chronic Conditions
|
Apr 15, 2020 |
|
A One-to-Many Approach to Gene Therapy for Retinal Disease
|
Apr 08, 2020 |
|
Targeting an Immune Switch in Rare Diseases
|
Apr 01, 2020 |
|
Targeting the Regulatory Genome to Treat Rare Blood Diseases
|
Mar 25, 2020 |
|
A Reborn Forma Therapeutics Focuses on Rare Blood Diseases and Cancer
|
Mar 18, 2020 |
|
Targeting Genetic Diseases at Their Root Cause
|
Mar 11, 2020 |
|
Targeting Hormone Dysregulation in Prader-Willi Syndrome
|
Mar 04, 2020 |
|
Rare Disease Advocates Take to the Hill
|
Feb 25, 2020 |
|
Targeting a Common Link in Otherwise Different Rare Diseases
|
Feb 19, 2020 |
|
Bringing Clinical Trials to Patients
|
Feb 12, 2020 |
|
Crowdsourcing Therapeutic Approaches for an Ultra-Rare Disease
|
Feb 05, 2020 |
|
Delivering Oligonucleotide Therapies to Any Cell Type
|
Jan 29, 2020 |
|
Protalix Looks Beyond Biobetters
|
Jan 22, 2020 |
|
Driving N-of-1 Therapies for Ultra-Rare Disease Patients
|
Jan 15, 2020 |
|
Cutting the Time and Cost of Drug Discovery with MicroOrgans
|
Jan 08, 2020 |
|
Acquisition Combines Two Pipelines for Rare Skin Diseases
|
Jan 02, 2020 |
|
Programming Cells to Produce Neuroprotective Factors to Treat ALS
|
Dec 26, 2019 |
|
Origin's Rapid Path to Seek Approval for Treatment of Ultra-Rare Metabolic Condition
|
Dec 18, 2019 |
|
Developing a Common Language for Rare Diseases
|
Dec 11, 2019 |
|
Why Payers Lag Drug Developers and Regulators in Embracing Real-World Evidence
|
Dec 04, 2019 |
|
How a Brother's Love Is Forging a Path for Customized Therapies for Rare Disease Patients
|
Nov 27, 2019 |
|
Addressing the Barriers to Accessing Experimental Therapies
|
Nov 20, 2019 |
|
A Journey to Advocacy
|
Nov 13, 2019 |
|
Turning Chronic Illness into a Fashion Statement
|
Nov 06, 2019 |
|
Connecting Young Adults with Rare and Chronic Conditions
|
Oct 30, 2019 |
|
Helping Caregivers Care for Themselves
|
Oct 23, 2019 |
|
A Festival of Moving Pictures
|
Oct 16, 2019 |
|
Deep Genomics Boasts an AI First in Drug Discovery
|
Oct 09, 2019 |
|
How to Live with a Disability with Your Mojo Intact
|
Oct 03, 2019 |
|
Why a Big Pharma Exec Embraced Rare Neurological Diseases
|
Sep 25, 2019 |
|
Fighting for Other People's Children
|
Sep 18, 2019 |
|
A Doctor's Quest to Cure His Own Rare Disease
|
Sep 11, 2019 |
|
Susannah Cahalan and her Month of Madness
|
Sep 04, 2019 |
|
The Benefits Support Groups Offer Rare Disease Patients
|
Aug 29, 2019 |
|
Targeting the Epigenetic Drivers of Cancer
|
Aug 21, 2019 |
|
Tackling Kidney Diseases with Orally-Delivered Enzymes
|
Aug 14, 2019 |
|
Giving Voice to Patients through Data
|
Aug 08, 2019 |
|
Seeking to Halt and Reverse Fibrotic Diseases
|
Jul 31, 2019 |
|
Addressing the Complexities of Rare Disease with Centers of Excellence
|
Jul 24, 2019 |
|
Using Cell Therapies to Restore Power in Mitochondrial Diseases
|
Jul 17, 2019 |
|
Passage Bio Betting on Penn Connection to Give It an Edge in Developing Gene Therapies
|
Jul 10, 2019 |
|
Finding Appropriate Endpoints and Measures for Rare Eye Diseases
|
Jul 03, 2019 |
|
Marrying Antibodies and Oligonucleotides to Target Genetic Medicine
|
Jun 26, 2019 |
|
What Rare Disease Patients Should Understand about Genetics
|
Jun 19, 2019 |
|
Program Seeks to Drive Collaborative Research Model Among Rare Disease Groups
|
Jun 12, 2019 |
|
A Gene Hunter Becomes a Drug Hunter Too
|
Jun 05, 2019 |
|
The Psychological Toll Rare Diseases Can Have on Healthy Siblings
|
May 29, 2019 |
|
Blood Test Can Provide Earlier Diagnosis of Autism Spectrum Disorder
|
May 22, 2019 |
|
Understanding the Role of Natural History Studies in Clinical Trials
|
May 15, 2019 |
|
How Two Mothers Discovered That a Drug for One Rare Disorder Might Benefit Another
|
May 08, 2019 |
|
Shortening the Diagnostic Odyssey
|
May 01, 2019 |
|
Eliminating Barriers to Adoption of Children with Rare Diseases
|
Apr 24, 2019 |
|
A Young Rare Disease Advocate Discusses Life with an Undiagnosed Condition
|
Apr 17, 2019 |
|
The Benefits of an Open-Source Registry for Rare Diseases
|
Apr 10, 2019 |
|
How a Patient Group Helped Drive Drug Development in Rett Syndrome
|
Apr 03, 2019 |
|
How Misperceptions Can Create Barriers to Care
|
Mar 27, 2019 |
|
Targeting Repeat Expansion Disorders with Next-Gen Antisense Drugs
|
Mar 21, 2019 |
|
Getting Misfolding Proteins to Shape Up
|
Mar 13, 2019 |
|
A Different Approach to Inhibiting the Complement System
|
Mar 06, 2019 |
|
Looking Back in Time to Find Rare Disease Patients Today
|
Feb 27, 2019 |
|
Ionis CEO Stanley Crooke Discusses Success as a Platform Technology Company
|
Feb 20, 2019 |
|
Orchard Looks toward Harvesting Expanded Gene Therapy Pipeline
|
Feb 13, 2019 |
|
Connecting Rare Disease Patients in India and the United States
|
Feb 06, 2019 |
|
Regenerative Medicine Moves into the Spotlight
|
Jan 30, 2019 |
|
Learning New Tricks from Pathogenic Bacteria to Target the Microbiome
|
Jan 24, 2019 |
|
Orphan Drug Accelerator Spin-Out Tackles Rare Endocrine Disorders
|
Jan 16, 2019 |
|
Aspa Therapeutics Forges Gene Therapy for Rare Neurological Condition
|
Jan 09, 2019 |
|
How Patient-Led Collaborations Are Transforming Rare Disease Drug Discovery and Development
|
Jan 02, 2019 |
|
Sobi Expands Its Immunology Franchise with HLH Drug Approval
|
Dec 27, 2018 |
|
Learning to Thrive
|
Dec 19, 2018 |
|
Late-Stage Trial Underway for Treatment of Rare Obesity Disorder
|
Dec 12, 2018 |
|
RegenxBio Bets Its Gene Therapy Technology Provides Competitive Edge
|
Dec 05, 2018 |
|
The Potential for Digital Health Technologies to Transform Clinical Trials
|
Nov 28, 2018 |
|
Matching Volunteers to Rare Disease Organizations in Need
|
Nov 21, 2018 |
|
How One Rare Disease Organization Used Technology to Provide Greater Patient Insight to FDA
|
Nov 14, 2018 |
|
The Potentially Deadly Consequences of an Oversight in Newborn Screening Tests
|
Nov 07, 2018 |
|
Advancing a New Approach to Sickle Cell Disease
|
Oct 31, 2018 |
|
Conquering Life with a Rare Disease
|
Oct 24, 2018 |
|
Why Rare Disease Drug Developers Need Regular Interaction with the FDA
|
Oct 17, 2018 |
|
Making the Case for an FDA Rare Disease Center of Excellence
|
Oct 10, 2018 |
|
An Elite Runner Makes a Cross-Canada Trek for His Son and Rare Disease
|
Oct 03, 2018 |
|
How Rare Disease Advocates Can Get The Most out of Collaborations
|
Sep 26, 2018 |
|
Establishing Clinical Standards for a Rare Disease
|
Sep 19, 2018 |
|
Teen Rare Disease Advocate Fights the Condition That Took His Brother's Life
|
Sep 12, 2018 |
|
Addressing the Emotional Toll of Rare Diseases
|
Sep 05, 2018 |
|
Forging a Research Agenda for a Rare Disease
|
Aug 29, 2018 |
|
Living with an Uncertain Rare Diagnosis
|
Aug 22, 2018 |
|
A Rare Disease Advocate Teaches Doctors to See and Speak Differently
|
Aug 17, 2018 |
|
The Caregiver Who Cried in the Shower
|
Aug 08, 2018 |
|
Curating Patient Data to Accelerate Rare Disease Drug Development
|
Aug 01, 2018 |
|
Forging a Research Agenda for a Rare Disease
|
Jul 25, 2018 |
|
Making the Journey from All-American Athlete to Rare Disease Advocate
|
Jul 18, 2018 |
|
How Destiny Lamonte Learned to Advocate for Herself and Others
|
Jul 11, 2018 |
|
Where the NIH Does Its Banking
|
Jul 05, 2018 |
|
Making Sense of Nonsense Mutations
|
Jun 27, 2018 |
|
Rewriting Faulty RNA
|
Jun 20, 2018 |
|
Experimental Drug for "Elephant Man" Disease May Benefit Certain Cancers Too
|
Jun 13, 2018 |
|
Programming Stem Cells to Protect Neurons in ALS and other Neurodegenerative Conditions
|
Jun 06, 2018 |
|
Why a Topical, Off-the-Shelf, Gene Therapy May Hold Promise for a Rare Skin Condition
|
May 30, 2018 |
|
An Ironman Champion with Cystic Fibrosis Discusses her Changing Relationship with Her Disease
|
May 23, 2018 |
|
Why an Experimental Fragile X Drug May Treat a Range of Neurodegenerative Diseases
|
May 16, 2018 |
|
The Case for a Daily Self-Injection to Treat Hemophilia
|
May 09, 2018 |
|
Addressing the Challenges of R&D for Rare Cancer Therapies
|
May 02, 2018 |
|
Learning to Communicate and Collaborate as a Patient Advocate
|
Apr 25, 2018 |
|
Seeking Answers for Undiagnosed Patients
|
Apr 18, 2018 |
|
When "Undiagnosed" Is Your Diagnosis
|
Apr 11, 2018 |
|
The Decision to Get Tested for Huntington's Disease and How It Changed Seth Rotberg's Life
|
Apr 04, 2018 |
|
Nonprofit Sees Need in Developing Gene Therapies for Inherited Eye Disesases
|
Mar 29, 2018 |
|
Casting a Wider Net for Genetic Diseases
|
Mar 21, 2018 |
|
Corbus Seeks to Quiet the Immune System in Chronic Conditions
|
Mar 14, 2018 |
|
Addressing the Challenges of Rare Disease Drug Development
|
Mar 07, 2018 |
|
Giving Voice to Patients at the FDA
|
Feb 28, 2018 |
|
Finding a Rare Friend
|
Feb 21, 2018 |
|
Why a Sports Physical May Trigger a Doctor to Think About a Rare Diagnosis
|
Feb 14, 2018 |
|
UPenn's Orphan Disease Center Takes a Rare Approach
|
Feb 07, 2018 |
|
How Patients Can Accelerate Translational Research in Rare Disease
|
Jan 31, 2018 |
|
Notre Dame Center Leverages Commonalities of Rare and Neglected Diseases
|
Jan 24, 2018 |
|
GalXC Quest: Dicerna Bets on Its Unqiue Approach to RNAi
|
Jan 17, 2018 |
|
Pharnext Takes a Unique Twist on Repurposing Drugs for Rare Diseases
|
Jan 10, 2018 |
|
uniQure Seeks to Find Elusive Market Success with Hemophilia B Gene Therapy
|
Jan 03, 2018 |
|
Savara Advances Pipeline of Inhaled Therapeutics for Rare Lung Diseases
|
Dec 27, 2017 |
|
Why the Microbiome May Provide a Novel Approach to Treating Certain Rare Diseases
|
Dec 20, 2017 |
|
FDA Outlines a Collaborative Approach for Pediatric Rare Disease Trials
|
Dec 13, 2017 |
|
Why Patient-Centred Outcomes Are Critical to Rare Disease Drug Trials
|
Dec 06, 2017 |
|
What the FDA's New Framework Means for Regenerative Therapies
|
Nov 29, 2017 |
|
Measuring the Value of Therapies for Ultra-Rare Diseases
|
Nov 22, 2017 |
|
Lessons from Pitching Venture Capitalists
|
Nov 15, 2017 |
|
Health Canada Scraps Plans for Orphan Drug Framework
|
Nov 08, 2017 |
|
New Investment Boosts Rare Disease Drug Accelerator
|
Nov 01, 2017 |
|
Understanding Rare Disease Caregiver Needs
|
Oct 25, 2017 |
|
Deriving Rare Disease Therapies from Human Plasma
|
Oct 18, 2017 |
|
Learning to Live with Purpose
|
Oct 11, 2017 |
|
A Mother's Race to Find a Treatment for Her Daughter's Ultra-Rare Condition
|
Oct 04, 2017 |
|
Screening for Rare Diseases
|
Sep 27, 2017 |
|
A Look at the Changing World of Work for People with a Chronic Illness
|
Sep 20, 2017 |
|
Aligning Interests in Rare Disease Partnerships
|
Sep 13, 2017 |
|
A Veteran Venture Capitalist Discusses Investing in Rare Disease Drug Development
|
Sep 06, 2017 |
|
A Patient Group Crafts Guidelines for Working with Pharma
|
Aug 30, 2017 |
|
Bringing Innovation to Business Models for Rare Disease Drug Development
|
Aug 23, 2017 |
|
Rethinking Accessibility and Dependency
|
Aug 16, 2017 |
|
How Simon Wheatcroft, Blinded by a Rare Disease, Became an Ultramarathoner
|
Aug 09, 2017 |
|
Spark Hoping Its Luxturna Will Become First FDA-Approved Gene Therapy
|
Aug 02, 2017 |
|
What Rare Patient-Investors Can Learn From Venture Capitalists
|
Jul 26, 2017 |
|
Novel Effort Delivers Failed DMD Drug to Former Clinical Trial Participants
|
Jul 19, 2017 |
|
Centogene Seeks to Deliver Diagnoses to Rare Disease Patients Early
|
Jul 12, 2017 |
|
Albireo Advances Pipeline for Rare Liver and Gastrointestinal Diseases
|
Jul 05, 2017 |
|
aTyr Pharma Seeks to Modulate Activity in FSHD and Other Rare Disease
|
Jun 28, 2017 |
|
Summit Pursues A Different Approach to Treating Duchenne
|
Jun 21, 2017 |
|
Delivering Enzyme Replacement Therapies Across the Blood-Brain Barrier
|
Jun 14, 2017 |
|
Sangamo Advances Gene Editing Therapies for Multiple Rare Diseases into the Clinic
|
Jun 07, 2017 |
|
Navigating the Challenges of Rare Disease Drug Development
|
May 31, 2017 |
|
Accelerating Rare Disease Drug Discovery
|
May 24, 2017 |
|
Experimental Rare Disease Therapy Wins New FDA Reg Med Designation
|
May 17, 2017 |
|
How Rare Disease Patients Can Get their Voices Heard in Drug Discovery and Development
|
May 10, 2017 |
|
One Patient's Difficult Road to a Rare Diagnosis
|
May 03, 2017 |
|
How Researchers Diagnosed Four Patients with a Never-Before-Identified Rare Disease in a Day
|
Apr 26, 2017 |
|
Stem Cell Gene Therapy Restores Immune System in Kids with SCID
|
Apr 19, 2017 |
|
Rare Disease Advocates Push for Incentives to Drugmakers Pulled from Cures Act
|
Apr 12, 2017 |
|
Non-Profit Drug Company Seeks to Advance Rare Disease Therapies
|
Apr 05, 2017 |
|
Innovative Collaboration Model Drives Treatment for Rare Disease
|
Mar 29, 2017 |
|
Girl with Rare Disease Inspires Search for Bone Marrow Donors
|
Mar 22, 2017 |
|
A Quest for Tribe: Searching for Others with the Same Ultra-Rare Mutation
|
Mar 15, 2017 |
|
Raising Awareness in a Town with a History of a Rare Disease
|
Mar 08, 2017 |
|
Helping Rare Disease Patients Navigate the World of Information
|
Mar 01, 2017 |
|
Overcoming the Challenges of Rare Disease Drug Development
|
Feb 22, 2017 |
|
Moving Rare Disease Therapies from Hope to Reality
|
Feb 15, 2017 |
|
Children’s National Launches First-of-its-Kind Rare Disease Center
|
Feb 08, 2017 |
|
A Common Link in a Group of Rare Diseases Creates an Opportunity for Repurposing
|
Feb 01, 2017 |
|
Sobi Works to Expand Rare Disease Indications for Its Drugs
|
Jan 25, 2017 |
|
Why Rare Disease Patients Should Work Together to Address Common Concerns
|
Jan 18, 2017 |
|
Solving Medical Mysteries with the Wisdom of Crowds
|
Jan 11, 2017 |
|
Embracing Collaborations to Drive Development of Rare Disease Therapies
|
Jan 04, 2017 |
|
Determining If a Child's Neurologic or Psychiatric Symptoms are Caused by an Infection
|
Dec 28, 2016 |
|
Helping Patients with the Same Undiagnosed Genetic Condition Find Each Other
|
Dec 21, 2016 |
|
An Entrepreneur Uses a Silicon Valley Approach to Tackle Rare Disease
|
Dec 14, 2016 |
|
Looking for Love with a Rare Disease
|
Dec 07, 2016 |
|
Why Genomics May Be in Google's DNA
|
Nov 29, 2016 |
|
The Power of Small Grants to Make Big Impacts
|
Nov 23, 2016 |
|
Building an International Registry for Rare Diseases
|
Nov 16, 2016 |
|
Discovering the Creative Use of Outrage
|
Nov 09, 2016 |
|
Diagnosing Rare Diseases with Facial Analysis
|
Nov 02, 2016 |
|
Charles River Sees Opportunity in Fostering Rare Disease Collaborations
|
Oct 27, 2016 |
|
Abeona Advancing Pipeline of Gene Therapies for Rare Diseases
|
Oct 19, 2016 |
|
A Patient Attends "an Academic Conference for Everyone"
|
Oct 12, 2016 |
|
Teen Advocate Preaches Message of Kindness
|
Oct 06, 2016 |
|
A Controversial Approval for a Duchenne Drug and What's Ahead
|
Sep 28, 2016 |
|
Matchmaker Exchange Helps Rare Disease Community Find Me a Find
|
Sep 21, 2016 |
|
The Promise of RNAi for Hereditary Angioedema and Other Rare Diseases
|
Sep 14, 2016 |
|
Why Natural History Studies Matter to Rare Disease Patients
|
Sep 07, 2016 |
|
Harnessing Big Data to Work for Rare Disease Patients
|
Aug 31, 2016 |
|
Lessons from Autism on Accelerating Drug Development
|
Aug 24, 2016 |
|
How One Rare Disease Group Leverages Relationships with Industry
|
Aug 17, 2016 |
|
Ice Bucket Challenge Begins to Yield Results
|
Aug 10, 2016 |
|
Why a Common Diabetes Drug May Be Able to Treat a Rare Disease
|
Aug 03, 2016 |
|
How to Improve Patient Access to Experimental Drugs
|
Jul 27, 2016 |
|
Using Social Media to Find Rare Disease Patients for Clinical Trials
|
Jul 20, 2016 |
|
How Rare Disease Patients Weigh Risks and Benefits of a Therapy
|
Jul 13, 2016 |
|
Why Homology Medicines Might Be a Game Changer for Gene Therapy
|
Jul 06, 2016 |
|
Orchard Hopes to Bear the Fruit of Gene Therapy
|
Jun 28, 2016 |
|
Novel Approach to Treat Rare Disease May Lead to Drugs for Common Ones
|
Jun 22, 2016 |
|
Brining Whole Genome Sequencing into the Clinic
|
Jun 15, 2016 |
|
Bringing Patient-Centricity to Clinical Trials
|
Jun 08, 2016 |
|
Determining If a Child's Neurologic or Psychiatric Symptoms are Caused by an Infection
|
Jun 01, 2016 |
|
Making Genetic Data Accessible to Researchers
|
May 25, 2016 |
|
How New Sequencing Technology Is Changing Our Understanding of Rare Diseases
|
May 18, 2016 |
|
Marathon Readies DMD Drug for FDA
|
May 11, 2016 |
|
Searching for Genes They May Protect Against Deadly Diseases
|
May 04, 2016 |
|
Helping Patients with the Same Undiagnosed Genetic Condition Find Each Other
|
Apr 27, 2016 |
|
Living with a Disease in Search of a Name
|
Apr 20, 2016 |
|
New Means of Regulating Genes May Hold Promise for Rare Disease Patients
|
Apr 13, 2016 |
|
Invitae Seeks a Faster, Cheaper Path to Diagnosing Genetic Diseases
|
Apr 06, 2016 |
|
Filmmaker Turns Lens on the Rare Disease Community
|
Mar 30, 2016 |
|
The Case for Keeping Orphans Where They Can Thrive
|
Mar 23, 2016 |
|
Rare Disease Legislation Advancing at National, State Level
|
Mar 16, 2016 |
|
The Evolving Relationship Between Patient Advocates and Pharma
|
Mar 09, 2016 |
|
Phil Reilly on The Quest to Save Children with Rare Genetic Disorders
|
Mar 02, 2016 |
|
Unlocking the Genetic Mysteries of Rare Diseases
|
Feb 24, 2016 |
|
A Vision for Making Kansas City a Rare Disease Center of Excellence
|
Feb 17, 2016 |
|
A Push in Canada for a National Rare Disease Strategy
|
Feb 10, 2016 |
|
Jazzed in Utah for World Rare Disease Day
|
Feb 03, 2016 |
|
Group Aims to Accelerate Repurposing of Drugs to Treat Rare Diseases
|
Jan 27, 2016 |
|
Gene Therapies Bring New Hope to Rare Disease Patients
|
Jan 20, 2016 |
|
Why Rare Disease Research Should Matter to Everyone
|
Jan 13, 2016 |
|
Researchers Say Reform of the Orphan Drug Act is Needed
|
Jan 06, 2016 |
|
Looking for Love with a Rare Disease
|
Dec 30, 2015 |
|
Gene Editing Tool Holds Promise in Rare Disease Treatments
|
Dec 23, 2015 |
|
A Cross Country Quest for Marrow Donors
|
Dec 16, 2015 |
|
Building Strength by Sharing Stories
|
Dec 09, 2015 |
|
North Carolina Seeks Leadership Role in Combating Rare Diseases
|
Dec 02, 2015 |
|
The Power of Small Grants to Make Big Impacts
|
Nov 25, 2015 |
|
Tearing Down Differences with Film
|
Nov 20, 2015 |
|
Educating Docs to Improve Diagnosis and Treatment of Genetic Metabolic Diseases
|
Nov 13, 2015 |
|
Providing Young Adult Cancer Patients and Survivors Resources They Need
|
Nov 05, 2015 |
|
Gene Therapy Company Born from Mother's Quest to Cure Daughter
|
Oct 30, 2015 |
|
Accelerating Rare Disease Research through Collaboration
|
Oct 23, 2015 |
|
Cracking the Genetics of Rare Diseases through Crowdsourcing
|
Oct 16, 2015 |
|
Teen Advocate to World: "We are More than Our Diseases"
|
Oct 09, 2015 |
|
Why the 21st Century Cures Act May Be in Trouble in the Senate
|
Oct 02, 2015 |
|
How Lisa Bentley Became a World Class Triathlete with Cystic Fibrosis
|
Sep 25, 2015 |
|
Understanding the Rare Disease Caregiver
|
Sep 17, 2015 |
|
Addressing Life-Threatening Rare Diseases with Gene Therapy
|
Sep 11, 2015 |
|
Living with, Rather Than for, a Rare Disease
|
Sep 04, 2015 |
|
An Entrepreneur Uses a Silicon Valley Approach to Tackle Rare Disease
|
Aug 28, 2015 |
|
Chronicling the Genomic Revolution
|
Aug 21, 2015 |
|
How a Radio Personality Became a Rare Disease Advocate
|
Aug 14, 2015 |
|
Why Genomics May Be in Google's DNA
|
Aug 07, 2015 |
|
An FDA Perspective on Rare Diseases
|
Jul 31, 2015 |
|
Helping Patients Take Control of Their Healthcare
|
Jul 24, 2015 |
|
Living Life Beyond Limits
|
Jul 17, 2015 |
|
Noah Coughlan Completes His 3,000 Mile Trek for Rare Diseases
|
Jul 09, 2015 |
|
How the 21st Century Cures Act Puts Patients at the Table
|
Jul 02, 2015 |
|
Using Cell and Gene Therapies to Treat Rare Skin Diseases
|
Jun 25, 2015 |
|
Incentives Boost Innovation in Rare Disease Therapies
|
Jun 19, 2015 |
|
Tensions Brew Between Pharma and Patient Advocacy Groups
|
Jun 12, 2015 |
|
The Attraction of Being a Rare Disease Drug Company
|
Jun 05, 2015 |
|
Confronting Genetic Destiny in The Lion's Mouth Opens
|
May 29, 2015 |
|
Royal Pains Throws Spotlight on Rare Diseases
|
May 22, 2015 |
|
Making the Case Rare Diseases are Fundamental
|
May 15, 2015 |
|
Programming the Immune System to Produce Drugs
|
May 08, 2015 |
|
How One Everyday Superhero Combats Rare Disease
|
May 01, 2015 |
|
Terminally Ill Patients Find Hope in Utah's Right to Try Law
|
Apr 24, 2015 |
|
Discovering the Creative Use of Outrage
|
Apr 17, 2015 |
|
New Diagnostic Offers Fast Answers For Group of Rare Diseases
|
Apr 10, 2015 |
|
One Researcher's Effort to Address a Fundamental Challenge in Rare Disease Research
|
Apr 03, 2015 |
|
Turning Lights and Camera into Action: A Mom's Search for Answers
|
Mar 27, 2015 |
|
A Teenager's Transformation from Patient to Advocate
|
Mar 20, 2015 |
|
How the Orphan Drug Act Reshaped the Rare Disease Landscape
|
Mar 13, 2015 |
|
Why the OPEN ACT Could Greatly Expand Treatments for Rare Disease
|
Mar 06, 2015 |
|
Rare Disease Company Seeks 100 Drugs in 10 Years
|
Feb 27, 2015 |
|
A Run Across America to Raise Rare Disease Awareness
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Feb 20, 2015 |
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Tapping the Public to Keep Scientists Current on Rare Disease Research
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Feb 13, 2015 |
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Why Raising Awareness Matters in the Fight Against Rare Diseases
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Feb 06, 2015 |
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The First Lady's Date who Embodied Precision Medicine
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Jan 29, 2015 |
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Startup with NIH Deal Seeks to Speed Drugs for Rare Diseases
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Jan 23, 2015 |
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Expanding the Potential of Crowdfunding
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Jan 16, 2015 |
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A Father's Search to Cure His Son's Rare Disease
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Jan 09, 2015 |
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How Venture Philanthropy Can Accelerate Drug Development
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Dec 31, 2014 |
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How New Funding Models Are Accelerating Drug Development
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Dec 04, 2014 |