You are using an unsupported browser. Please update your browser to the latest version on or before July 31, 2020.


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