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2013 RARE Patient Advocacy Summit
Advances in Rare Immunological Diseases
Breakthroughs for Rare Neurological Syndromes
Building Valuable Registries and Natural History Studies
Capacity Building Strategies: Managing Volunteers
Challenge Your Limits
Changing Policy, Saving Lives
Chasing Cures: The Power of Patients
Clinical Care: Building Standards of Care for Your Disease Community
Closing Session - Data and Technological Innovation
Collaboration Among Foundations
Crafting a Career: Making a Fulfilling Professional Life with a Rare Disease
Data, Data, Data: Sharing and Owning Data to Game Change Rare Disease Research
Day 1: Track 1, Caring for Yourself as Caregiver, Speakers: Grace Whiting, J.D., Kathleen Kelly
Day 1: Track 3, Building an Engaged Patient Community, Speakers: Sue Kahn, Blake Sheweyyy
Day 1: Track 4, Rare Idiopathic Disorders, Jeanette McCarthy, M.P.H., Ph.D., Caroline Loewyy
Day 2: A Call to ACTION for #CuresNOW, Speakers: Max Bronstein, Tracy VanHoutan, Melissa J. Hogan
Day 2: Track 1, Developing Volunteer Leaders, Speakers: Louise Vetter, Eveline Honig
Day 2: Track 3, Understanding Expanded Access, John Lagus, Steve Walker, Kristina Broadbeltlt
Day 2: Track 4, Genetic Counselors, Willonie Mendonca,M.Sc., M.S., LCGC, Erica Ramos, M.S., LCGC
Developing Partnerships Among Rare Communities and Individuals
Early Drug Development What Patients Bring
Eating Well Taking Care of Yourself through Nutrition
Family Planning: Decisions and Considerations
Finding Answers: The Importance of Communicating with Clinicians
Fundraising Ideas Anyone Can Try
Genome Sequencing: Hope, Promises and Limitations
Is the Research Plan Working? Evaluation and Accountability for Research Grants and Contracts
Leveraging Digital Tools for Reach and Impact
Looking for Answers in Uncertainty: Managing When There is No Diagnosis or Prognosis
Medical and Scientific Advisory Board Presents Patients Driving Advances From Research to Therapy
Modern Therapies for Emotional Health: Different Ways to Cope
Next: Imagining the Future of Rare Disease
Patients and Advocates as Entrepreneurs: New Models for Drug Development
Patients and Caregivers: Shaping Clinical Drug Development
Rachel Callander Welcome and Keynote
RARE Champion of Hope Awards Celebration
Rare Disease Research: Getting Your Disease Noticed
RARE on the Road 2021: A Rare Disease Leadership Interactive Webinar
RARE Patient Advocacy Summit
Rare Research Resources: Animal Models and 3D Engineered Tissues
Rare Research Resources: Biomarkers
Rare Research Resources: CRISPR System and iPS Cells
Rare Therapies: A New Era for Genetic Medicines
Rare Therapies: Pharmacological Chaperones and Cell Therapies
Romance While Rare
Searching for A Diagnosis: Colleen Olson
Setting Up Your RARE Portal
Summit Day 1: Innovations in Science Brief- American MedChem, Robert Selliah, PhD
Summit Day 1: Innovations in Science Presentation Brief 1- RARE Science, Christina Waters, PhD, MBA
Summit Day 1: Opening Remarks and Session 1: Caregivers- Strategies to stay Afloat
Summit Day 1: Session 2- E-Patient Revolution
Summit Day 1: Session 3- Shaping Regulatory Policies That Put Patients First
Summit Day 2: Innovations In Science Presentation Brief 4- Immunosoft, Matthew Scholz, PhD
Summit Day 2: Innovations in Science Presentation Brief- Project Violet, Jim Olsen, PhD
Summit Day 2: Opening Remarks
Summit Day 2: Session 4- Deep Dive, Mobilizing Your Community For Patient-Focused Drug Development
Summit Day 2: Session 4- Patient-Centered Benefit-Risk Assessment: Why It Matters To You
Summit Day 2: Session 5- Deep Dive: Missions, Goals and Strategic Planning- Keegan Johnson
Summit Day 2: Session 5- The Unstoppable Charity
Summit Day 2: Session 6- Transition And Transformation- Rare Disease In Adolescence And Adulthood
Summit Day 2: Session 7- Preparing for Success- Lobbying At The State And Federal Level
Summit Day 2: Session 8- Putting Into Practice- 21st Century Cures
Susannah Cahalan - 2019 RARE Patient Advocacy Summit Keynote
Sustaining an Organization: Managing Long-Term Growth and Development
Technologies for Learning about Rare Disease from Patients and Caregivers
The Future of Rare Metabolic Syndromes
The New Normal: Patient Communities Drive Innovation
The Nuts and Bolts of Collaborations: Be Prepared for the Paperwork
The Secret Sauce for Forward Movement in Research and Scientific Collaborations
The Value of Rare Disease Therapies: Patient Perspectives Needed
THRIVE: Game Changing the World of Rare Disease through Collaboration
Track 1: Beyond Diagnosis Working with a Genetic Counselor During Your Rare Journey
Track 1: Coping with a Long Term Hospital Stay
Track 1: Home Team Advantage How to Arrange for Palliative Care
Track 1: RARE Kids Growing Up Experiencing Transitions in Care and Life
Track 1: RARE Kids In Schools The Opportunities and Challenges
Track 1: Support for the Rare Disease Family Caring for Siblings of Kids with Rare Diseases
Track 1: The Road Already Traveled Translating your Experience into Support for New Families
Track 2: Advocacy for the Community Newborn Screening
Track 2: A New Approach for State Advocacy Rare Disease Advisory Councils
Track 2: Building and Sustaining a Rare Disease Non Profit The Importance of Developing A Strategic
Track 2: Fresh Minds, New Ideas Working with the Next Generation of Clinicians and Researchers
Track 2: Funding Sources You May Not Have Heard Of Funding and Collaboration Opportunities
Track 2: Patient Advocacy An Update on Federal Legislation
Track 2: The Bottom Line on Giving Research Grants
Track 2: The Promise and Perils of Working with Industry Lessons from Advocacy Group Experience
Track 3: Getting from the Lab to the Clinic Faster Advancing Translational Science
Track 3: How a Therapy Gets Developed and Approved The R&D Roadmap and the Growing Role of Patients
Track 3: Incorporating "Real World Evidence" into Drug Development and Approval
Track 3: Laying the Foundation The Role and Goals of Discovery and Preclinical Research
Track 3: Opening Keynote - Adaptability in the Face of Adversity/ MSAB Presents
Track 3: Patients as Investors
Track 3: Show Your Data What Do You Need To Have Before Approaching Industry
Track 3: The Drug Re discovery Pipline Finding New Indications for Approved Therapeutics
Track 3: Understanding the Constraints on Clinical Trials Inclusion Criteria, Control Groups, Blind
Track 3: Working with NCATS/ EveryLife Advocacy Outreach/ Digital Health
Track 4: Artificial Intelligence Making Research and Diagnosis Faster and More Accurate
Track 4: Bringing Back What Is Missing Enzyme Replacement Therapies for Rare Disease
Track 4: Improving Quality of Life 3D Printing Assistive Devices
Track 4: iPS Cells A New Tool for Research and Drug Development
Track 4: Making Sense of Antisense and Other Oligonucleotide Therapies
Track 4: The Progress of Gene Therapy and Gene Editing
Track 4: The Promise of Regenerative Medicine Cell Therapy and Tissue Engineering
Track 4: Understanding the Biology of Rare Diseases
Transition of Care Planning for Care for Children with a Rare Disease
Understanding the Emotional Health of Rare Disease Families
Understanding the Emotional Health of Rare Disease Patients
Update from the Community: Caregiver Survey
Update from the Community: Patient-Focused Drug Development Meetings
Update from the Community: Quality of Life Markers
We Can’t Do it Alone: Structure and Governance for Research Collaborations
Welcome and Main Session: Rare Disease Updates from the Global Stage
What is Druggable New Therapeutic Targets in Rare Disease
Your Diagnosis Matters - What are ICD and ORPHA Codes and Why are They Important