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