Agenda

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    • Wednesday, 09/13/17
    •  
      3:00 PM  -  7:00 PM
      Registration & Help Desk
      Location: Pre-Function Foyer
      3:00 PM  -  7:00 PM
      Exhibitor Pavilion
      Location: Pre-Function Foyer
    • Thursday, 09/14/17
    •  
      6:00 AM  -  7:00 AM
      Sunrise Yoga
      Location: The Lawn
      Embrace your inner yogi and unroll your yoga mat for an invigorating full-body flow. This relaxing class is built for all levels and is the perfect way to kick off a weekend full of education and inspiration.
      6:30 AM  -  5:00 PM
      Registration & Help Desk
      Location: Pre-Function Foyer
      6:30 AM  -  8:00 AM
      Breakfast
      Location: Pre-Function Foyer

      Sponsored by:

      Illumina

      illumina-blue

      6:30 AM  -  5:00 PM
      Exhibitor Pavilion
      Location: Pre-Function Foyer
      6:30 AM  -  5:00 PM
      Attendee Networking Lounge
      Sponsored by:
      Alexion Pharmaceuticals

      alexion-blue

      8:00 AM  -  8:15 AM
      Welcome & Meeting Kickoff
      Location: Grand Ballroom ABC
      Speakers:
      8:15 AM  -  9:15 AM
      Opening Keynote Adaptability in the Face of Adversity
      Location: Grand Ballroom ABC
      Simon Wheatcroft has been inspiring, motivating, and sharing his vision of the adaptability of technology and the mind to achieve the impossible all over the world. Join us to hear his heartening story to kick-off our two days to connect, collaborate, and activate!
      Speakers:
      9:15 AM  -  10:15 AM
      MSAB Presents A Patient's Guide to Next-Generation Sequencing Methods
      Location: Grand Ballroom ABC
      Genetic sequencing often plays a key role in diagnosis and research. New approaches may give sequencing an even more powerful role in disease identification, understanding, and treatment. In this session, leaders in next-generation sequencing will discuss different approaches such as whole exome sequencing, whole genome sequencing, and RNA-sequencing, and their significance for the rare disease community.
      10:15 AM  -  10:30 AM
      Morning Break
      Location: Pre-Function Foyer

      Sponsored by:

      Insmed Incorporated

      insmed-blue-square
       

      Track and Workshop Sessions - 10:30 AM

      10:30 AM  -  11:30 AM
      Track 1: Strengthening Your Care & Support Team
      Location: Grand Ballroom D

      Home Team Advantage: How to Arrange for Pallitive Care

       

      Palliative care is often misunderstood by patients and families. As a result, many families may never ask for the at-home support that can be a critical part of rare disease care. Palliative care can provide both help and hope for families striving to ensure the best care for their loved ones. Come hear about the role of palliative care for rare disease patients and practical strategies as to how to access these services.

       

      Sponsored by:

      Horizon Pharma

      horizon-logo-square

       

      10:30 AM  -  11:30 AM
      Track 2: Rare Disease Activism
      Location: Grand Ballroom E

      The Bottom Line on Giving Research Grants

       

      More and more, patient organizations are advancing research by developing and funding grant programs themselves. However the process is more complex than simply awarding funds to researchers. In this session, you will hear from patient advocates who have created successful research partnerships leading to advances in rare disease research. Learn about how to plan a research program, the nuances and expectations of working with researchers, and how to execute a research grant program.

       

      10:30 AM  -  11:30 AM
      Track 3: Drug Development Pathway
      Location: Grand Ballroom ABC

      How a Therapy Gets Developed and Approved: The R&D Roadmap and the Growing Role of Patients

       

      In rare disease, patients have been active partners in research and drug development for years. They have become foundational within the research lab, clinical trials, regulations, and clinic itself. Join both industry and patient innovators to learn about the approval pathway for new rare disease therapies and the many ways that patient voices can maintain a key role in the process.

       

      Sponsored by: 

      PRA Health Sciences

      pra

      Speakers:
      10:30 AM  -  11:30 AM
      Track 4: Science and Technology Innovation
      Location: Theatre

      Understanding the Biology of Rare Diseases

       

      Most rare diseases have a genetic cause. But identifying the gene or genes that underlie a rare disease is only the first step in understanding the disease. Once identified, determining whether the gene is involved in making a protein, moderating the amount of a protein, or impacting other proteins becomes integral to developing approaches in drug therapy with the greatest potential for efficacy. Join research leaders in rare disease for an overview of what is known about rare disease biology, and how it informs research and therapy development.

      10:30 AM  -  11:30 AM
      Workshop 1: Successful Scientific & Family Conferences
      Location: Saddleback

      How to Organize a Successful Scientific Conference

       

      Hosting a scientific conference can set the stage for research collaborations that can advance the understanding of rare diseases and R&D of therapeutics. But what details must you consider when planning such a conference, and what is the best approach to engage researchers? Hear from two patient advocate leaders who have been instrumental in putting together scientific conferences. Learn about identifying speakers and participants, extending invitations, approaching sponsors, and developing agendas to ensure a successful outcome for your scientific conference or event.

      Speakers:
      10:30 AM  -  11:30 AM
      Workshop 2: Beyond Clinical Care
      Location: Trabuco

      Difficult Discussions: How to Talk to Your Child About Their Degenerative Rare Disease  

       

       

      Is ignorance really bliss? In rare disease, we would argue it is not. Rather, knowledge is power, and the key to helping patients and families learn to cope, problem solve, and better manage their current and future health. Hear from experts and patients about how and when they engage in these often difficult discussions.

       

       

       In Partnership with: Child Neurology FoundationCNFXSm

      Speakers:
       

      Track and Workshop Sessions - 11:40 AM

      11:40 AM  -  12:40 PM
      Track 1: Strengthening Your Care & Support Team
      Location: Grand Ballroom D

      Beyond Diagnosis: Working with a Genetic Counselor During Your Rare Journey

       

      Genetic counselors play a critical role in the diagnosis of a rare disease. They can also offer important guidance about treatment options, how best to transition from pediatric to adult health care providers, and ways to develop a clinical care team. How can genetic counselors help you create ongoing support for patients and families within your disease community? How would you identify, approach, and engage them? Learn about ways to best gain support from these key members of your care team.

      Sponsored by:

      Horizon Pharma

      horizon-logo-square

       

      11:40 AM  -  12:40 PM
      Track 2: Rare Disease Activism
      Location: Grand Ballroom E

      Fresh Minds, New Ideas: Working with the Next Generation of Clinicians and Researchers

       

      The power of collaborations with researchers and clinicians is well-established. Initiating such collaborations in rare disease during the early stages of their respective careers is particularly advantageous. It promotes enthusiasm and innovation that drive funding for research and can propel careers. Join leaders from advocacy organizations who have developed programs that accelerate research in rare disease by supporting researchers during the early stages of their careers.

      11:40 AM  -  12:40 PM
      Track 3: Drug Development Pathway
      Location: Grand Ballroom ABC

      Laying the Foundation: The Role and Goals of Discovery and Preclinical Research

       

      Discovery and preclinical research are important in the early phases of drug research and development. How are targets identified for therapies and discovered for drug development? How does discovery research differ from preclinical research? And how do these efforts lay the groundwork for clinical research? Learn the answers to these questions from leaders in pharma research by attending this session.

      Sponsored by:

      PRA Health Sciences

      pra

      11:40 AM  -  12:40 PM
      Track 4: Science and Technology Innovation
      Location: Theatre

      iPS Cells: A New Tool for Research and Drug Development

       

      Induced pluripotent stem (iPS) cells are offering new, revolutionary approaches to discovery and therapy development for rare diseases. Researchers can take cells from a person's skin or blood, reprogram them into iPS cells, and grow them into liver, kidney, nerve, or any other cell type affected by a disease. With these iPS cells, researchers can model how a rare disease works or even screen or test potential therapies. Join experts and patient advocates to learn more about how iPS cells are becoming a critical research tool.

      11:40 AM  -  12:40 PM
      Workshop 1: Successful Scientific & Family Conferences
      Location: Saddleback

      Growing Your Scientific Network After a Research Conference

       

      Organizing and hosting a research conference is only one step in the process of actively steering research for rare disease. You can capitalize on the momentum generated by a scientific conference to solidify and grow your scientific network. Learn about how to continue to make connections in rare disease research, and extend your reach world-wide.

      11:40 AM  -  12:40 PM
      Workshop 2: Beyond Clinical Care
      Location: Trabuco

      Beyond Clinical Care: Working with an In-Home Nursing Caregiver Team

       

      Managing a child or loved one with a life-limiting illness is incredibly difficult. You can't do it all. Working with an in-home care team can help relieve some of the burden. This is a critical resource that provides important value, but also comes with the price of navigating the development of trust and a lack of privacy in your own home. Learn from patient advocates and experts about ways to access this vital resource and build strong interactions with a team of caregivers who become critical to the health of your loved one.

       
      12:40 PM  -  2:00 PM
      Networking Lunch
      Location: The Patio

      Sponsored by:

      Shire

      shire-square-logo

       

      Track and Workshop Sessions - 2:00 PM

      2:00 PM  -  3:00 PM
      Track 1: Strengthening Your Care & Support Team
      Location: Grand Ballroom D

      Coping with a Long-Term Hospital Stay

       

      A long-term hospital stay is difficult for patients and their families. But you can take action to plan for your financial needs, and work with hospital caregivers to make arrangements to care for your family. Such strategies can help keep patients engaged in life beyond the hospital. Hear from financial planning and child life experts about how to best plan for a long-term hospital stay.

      Sponsored by:

      Horizon Pharma

      horizon-logo-square

       

      Speakers:
      2:00 PM  -  3:00 PM
      Track 2: Rare Disease Activism
      Location: Grand Ballroom E

      Funding Sources You May Not Have Heard Of: Funding and Collaboration Opportunities with Diverse Federal and State Agencies

       

       There are several federal agencies that support rare disease research, as well as a diverse range of state agencies. But the first step in identifying funding sources is knowing about the existence of this support, and where to look. Join us for a discussion about the process of identifying both federal and state funding sources for rare disease research.

      2:00 PM  -  3:00 PM
      Track 3: Drug Development Pathway
      Location: Grand Ballroom ABC

      The Drug Re-positioning Pipeline: Finding New Indications for Approved and Shelved Therapeutics

       

       

      Drug development for new therapies is a long and expensive process. Repositioning drugs already approved by the FDA for another indication or that have been dropped from development by a company could be a way to develop rare disease therapies more quickly, efficiently, and less costly. Hear actual examples that showcase these important opportunities for patient investors, and find out if these options exist for your disease community.

      Sponsored by:

      PRA Health Sciences

      pra

      Speakers:
      2:00 PM  -  3:00 PM
      Workshop 1: Successful Scientific & Family Conferences
      Location: Saddleback

      How to Plan a Successful Family Conference

       

      It takes more than just an great venue to make a family conference successful. Successful family conferences require a tremendous amount of planning, and consideration of suitability of location, accommodations, onsite childcare, special programming, agenda, and of course, cost to attend. Join us for this workshop to learn important tips from patient advocates and planning experts to ensure that your next family experience is a meaningful and comfortable experience for both patients and families who attend.

      2:00 PM  -  3:00 PM
      Workshop 2: Beyond Clinical Care
      Location: Trabuco

      Beyond Clinical Care: Working with Occupational and Physical Therapists

       

      Occupational and physical therapy can serve a vital role in the care of rare disease patients. Come learn about these resources from a patient advocate, including how you might access them, what your plan of care should include, and practical tips for including occupational and physical therapists as part of your, or your loved one's, care team.

      Speakers:
       

      Track and Workshop Sessions - 3:15 PM

      2:00 PM  -  3:00 PM
      Track 4: Science and Technology Innovation
      Location: Theatre

      The Promise of Regenerative Medicine: Cell Therapy and Tissue Engineering

       

      Advances in science and technology have prompted the development of new therapeutics in rare disease. Approaches are being tested that use cells, or cells combined with other molecules, in order to correct the problem in tissues, or replace tissues entirely. Join this session for an insightful discussion about current clinical research and future opportunities in cell therapy and tissue engineering, and how these advances may impact your rare disease.

       
      3:00 PM  -  3:15 PM
      Afternoon Break
      Location: Pre-Function Foyer

      Sponsored by:

      Sanofi Genzyme 

      sanofi-genzyme-blue-square

       

      Track and Workshop Sessions - 2:00 PM

      3:15 PM  -  4:15 PM
      Track 4: Science and Technology Innovation
      Location: Theatre

      The Progress of Gene Therapy and Gene Editing

       

      The community is hopeful that research efforts are advancing closer toward curing rare diseases by correcting the underlying genetic causes. What is the status of developing gene therapy and gene editing approaches, and when might these be viable therapies to pursue for your rare disease? Industry leaders will discuss current and future efforts, opportunities, and roadblocks.

      Speakers:
       

      Track and Workshop Sessions - 3:15 PM

      3:15 PM  -  4:15 PM
      Track 1: Strengthening Your Care & Support Team
      Location: Grand Ballroom D

      The Road Already Travelled: Translating your Experiences into Support for New Families in Your Disease Community Featured content by the Child Neurology Foundation’s Family Support & Empowerment Program

       

      The caregiving experience can often be isolating. Yet it is important to remember that others in your community share the same feelings, ask the same questions, and would benefit from the same support. Drawing support and sharing information and helpful resources from others in the rare disease community can help you and your loved ones with diagnosis, treatment, and care. Come hear from a leader in The Child Neurology Foundation’s "Family Support and Empowerment Program" about this program of peers designed to ensure that no caregiver ever walks alone.

      Sponsored by:

      Horizon Pharma

      horizon-logo-square


      In Partnership with:

      Child Neurology Foundation

      CNFXSm

      Speakers:
      3:15 PM  -  4:15 PM
      Track 2: Rare Disease Activism
      Location: Grand Ballroom E

      The Promise and Perils of Working with Industry: Lessons from Advocacy Group Experiences on Addressing Potential Conflicts of Interest

       

      We know how critical it is for the patient community to become partners with organizations that are helping to develop treatments and cures for rare diseases. Developing proactive partnerships and working with biotech/pharma have become a necessity. This session will help you understand the importance of these partnerships. It will also help you design your own community's guidance document that states how, when, why, and what your engagement with these companies can be. This session will be particularly beneficial for growing organizations with potential therapies in development.

      Speakers:
      3:15 PM  -  4:15 PM
      Track 3: Drug Development Pathway
      Location: Grand Ballroom ABC

      Incorporating "Real-World Evidence" into Drug Development and Approval

       

      Collecting data is key in studying disease and evaluating therapeutics. Data sources can include registries, observational studies, and patient-centered outcomes. This session will describe evidence, outcomes, and data collection, and how they can influence drug development today and in the future.

       

      Sponsored by:

      PRA Health Sciences

      pra
      3:15 PM  -  4:15 PM
      Workshop 1: Successful Scientific & Family Conferences
      Location: Saddleback

      Building Community and More: The Full Potential of Family Conferences

       

      Family conferences offer the opportunity for rare disease families to come together and share experiences, feelings, and support. New and valuable information derived from family conferences can also provide attendees with a greater understanding of the disease, which can result in enhanced clinical care, or perhaps a new direction in research. In this session you can learn about a successful family conference model that allows attendees to realize the full potential of these vital gatherings.

      3:15 PM  -  4:15 PM
      Workshop 2: Beyond Clinical Care
      Location: Trabuco

      Beyond Clinical Care: Working with Educational Aides

       

      Children with rare diseases require additional care and support at school. Educational aides can help provide assistance in helping your child work towards meeting his or her educational goals. Come hear the experiences of a patient advocate and learn about the value of educational aides, how to seek their assistance, and ways to engage them in your child's educational journey.

      Speakers:
       
      4:30 PM  -  5:30 PM
      Stories of Hope and Persistance: We Carry Kevan
      Location: Grand Ballroom ABC

      Documentary Screening/ Post Screening Discussion

       

      Join us for a documentary screening and post-screening discussion to hear from Kevan Chandler, an amazing young man, and his friends, as they share information about their journey to various countries around the globe - all while overcoming the limitations of spinal muscular atrophy. "We Carry Kevan" is a story of innovation and perseverance which inspires us all to pursue our dreams.

      5:30 PM  -  7:30 PM
      Backyard BBQ
      Location: The Patio

      Come out to unwind and network with fellow attendees at our Backyard BBQ. Enjoy food and drink while playing a variety of fun interactive games in this one-of-a-kind RARE space.
       

      Sponsored by:

      PRA Health Sciences

      pra

    • Friday, 09/15/17
    •  
      6:00 AM  -  7:00 AM
      Sunrise Yoga
      Location: The Lawn

      Embrace your inner yogi and unroll your yoga mat for an invigorating full-body flow. This relaxing class is built for all levels and is the perfect way to kick off a weekend full of education and inspiration.

      6:30 AM  -  3:00 PM
      Registration & Help Desk
      Location: Pre-Function Foyer
      6:30 AM  -  8:00 AM
      Breakfast
      Location: Pre-Function Foyer

      Sponsored by:

      Amicus Therapeutics

      amicus-square

      6:30 AM  -  3:00 PM
      Exhibitor Pavilion
      Location: Pre-Function Foyer
      6:30 AM  -  3:00 PM
      Attendee Networking Lounge
      Location: Pre-Function Foyer
      Sponsored by:
      Alexion Pharmaceuticals
      alexion-blue


      8:00 AM  -  8:15 AM
      Welcome
      Location: Grand Ballroom ABC
      Speakers:
      8:15 AM  -  9:15 AM
      Catalyzing Translation: Working with the National Center for Advancing Translational Sciences
      Location: Grand Ballroom ABC
      Rare disease research depends on translational research, or the process of moving findings from the lab or from patient data into clinical research. The mission of the National Center for Advancing Translational Sciences (NCATS) at the NIH is to catalyze the development of innovations that accelerate this process, and includes several programs focused on rare disease. Learn about NCATS and how to engage with these innovative rare disease programs created to ensure that therapies are developed quickly.
      9:15 AM  -  9:30 AM
      Everylife Foundation Advocacy Outreach
      Location: Grand Ballroom ABC

      In Partnership With:

      Everylife Foundation

      EveryLifeLOGO_square

      Speakers:
      9:30 AM  -  10:15 AM
      How Digital Health Will Transform Rare Disease Clinical Trials
      Location: Grand Ballroom ABC
      Rapid advances in technology are affecting all parts of the rare disease R&D pathway. One area in which these innovations are rapidly being adopted is clinical trials. Clinical trials are evolving to incorporate advances in digital health technology to enable faster and more efficient patient recruitment, stronger patient engagement, the collection of a wide range of robust data on patient outcomes, and through the use of wearable devices and sensors. Leaders in digital health will discuss how these innovations are being realized to transform clinical trials.
      10:15 AM  -  10:30 AM
      Morning Break
      Location: Pre-Function Foyer

      Sponsored by:

      EMD Serono

      emd-square-blue

       

      Track and Workshop Sessions - 10:30 AM

      10:30 AM  -  11:30 AM
      Track 1: Care & Support for Your Kids
      Location: Grand Ballroom D

      Support for the Rare Disease Family: Caring for Siblings of Kids with Rare Diseases

       

      Siblings in rare disease families often find themselves missing much of their childhood, as they focus on helping their families as best as they can. Parents are faced with the need to ensure that they support the financial futures of all their children. Join a rare disease sibling and a financial planner to gain insights into the many gifts and challenges faced by siblings, and how to best ensure they are cared for.

       

      Sponsored by:

      Horizon Pharma

      horizon-logo-square

       

      10:30 AM  -  11:30 AM
      Track 2: Legislative & Medical Advocacy
      Location: Grand Ballroom E

      Building and Sustaining a Rare Disease Non-Profit: The Importance of Developing A Strategic Plan

       

      Strategic planning can be both exciting and frustrating. But planning is vital to the long-term viability and growth of any nonprofit organization. Learn about the purpose of a strategic plan, who should be involved in developing the plan, questions that should be addressed by the plan, and the key characteristics your plan should include.

      Speakers:
      10:30 AM  -  11:30 AM
      Track 3: Drug Development: Getting into the Clinic
      Location: Grand Ballroom ABC

      Getting from the Lab to the Clinic Faster: Advancing Translational Science

       

      The National Center for Advancing Translational Sciences is working to revolutionize biomedical research by developing innovations for the process of translational research, which refers to moving findings from the lab or data from populations, into testing of therapies. Join the Director of NCATS to learn more about how the Center is creating new partnerships and tools for discovery and preclinical research, and their impact on the rare disease community.

       

      Sponsored by: 

      PRA Health Sciences

      pra
      Speakers:
      10:30 AM  -  11:30 AM
      Track 4: Science and Technology Innovation
      Location: Theatre

      Making Sense of Antisense and Other Oligonucleotide Therapies

       

      New types of drugs are demonstrating immense promise for many rare diseases. Oligonucleotide therapies, which block the formation of a protein or restore the function of a protein, are moving into the spotlight as a way to target rare diseases that have been described as "undruggable". In this session, industry leaders will provide insights and updates on the potential of these therapies for rare disease.

      Speakers:
      10:30 AM  -  11:30 AM
      Workshop 1: Capacity Building for Your Organization
      Location: Saddleback

      Accounting and Compliance for Non-profits

       

      Once your organization has a nonprofit designation, there is significant work involved for accounting and maintaining your tax-exempt status. What do you need to do to ensure that your organization is compliant with federal and state laws and best practices? Hear from leaders in nonprofit management to learn about critical filings and activities to keep your organization thriving and in good standing.

      Speakers:
      10:30 AM  -  11:30 AM
      Workshop 2: Development and Approval of New Therapies
      Location: Trabuco

      The Rare Disease Organization as a Researcher: Understanding Institutional Review Board (IRB) Approval for Natural History Studies

       

      Natural history studies are a critical step in the research pathway for rare diseases. But these studies must be robust, well-designed clinical studies which follow federal regulations for clinical research. This means that the study must be approved by an Institutional Review Board (IRB). Join a parent advocate and researcher who will provide guidance as to the purpose and approach of an IRB, and the requirements to consider when reviewing your natural history study proposal.

      Speakers:
       

      Track and Workshop Sessions - 11:40 AM

      11:40 AM  -  12:40 PM
      Track 1: Care & Support for Your Kids
      Location: Grand Ballroom D

      RARE Kids In Schools: The Opportunities and Challenges

       

      Rare disease kids face many challenges in school, but they also can realize several opportunities. How do you ensure your child gets assistance from elementary school through higher education? What resources are available, and how do you access them? Leading advocates will provide insights and tips that that can help ensure that your child's education is a robust and valued experience.

       

      Sponsored by:

      Horizon Pharma

      horizon-logo-square

       

      11:40 AM  -  12:40 PM
      Track 2: Legislative & Medical Advocacy
      Location: Grand Ballroom E

      A New Approach for State Advocacy: Rare Disease Advisory Councils

       

      Many policies that affect the rare disease community emanate at the state level. There is now a growing movement to create advisory councils with a strong patient voice to research and recommend changes to state policies and organizations that will help the rare disease community. Key members of a state advisory council will lead you through the ins and outs of creating and managing a state rare disease advisory council.

      Speakers:
      11:40 AM  -  12:40 PM
      Track 3: Drug Development: Getting into the Clinic
      Location: Grand Ballroom ABC

      Show Your Data: What Do You Need To Have Before Approaching Industry

       

      What information is required by industry when weighing the addition of a therapy to its pipeline? What can rare disease patients and advocacy organizations do to provide the needed information to industry? Hear from industry leaders to know the key data your community can contribute when beginning partnering discussions with industry.

       

      Sponsored by:

      PRA Health Sciences

      pra

      Speakers:
      11:40 AM  -  12:40 PM
      Track 4: Science and Technology Innovation
      Location: Theatre

      Bringing Back What Is Missing: Enzyme Replacement Therapies for Rare Disease

       

      Enzymes are vital to numerous processes in our bodies. Many rare diseases are the result of a missing or non-functional enzyme. Giving patients the missing enzyme has been approved as a treatment for some rare diseases. In this session, leading industry experts and patient advocates will describe how enzyme replacement therapy works, and discuss future possibilities of this therapeutic approach.

      Speakers:
      11:40 AM  -  12:40 PM
      Workshop 1: Capacity Building for Your Organization
      Location: Saddleback

      How to Develop a Strategic Plan for Your Organization

       

      You know about the importance of a strategic plan for your organization, but how do you take amazing ideas and translate them into practical and measurable goals with attainable objectives to strengthen your organization? Where are the potential pitfalls in the process? Join us, as leaders in nonprofit management work through a case study that can help your organization plan for the future.

      Speakers:
      11:40 AM  -  12:40 PM
      Workshop 2: Development and Approval of New Therapies
      Location: Trabuco

      Building a Patient Registry that's Useful

       

      Creating a registry is a key first step in helping to advance research, and ultimately, the development of a treatment. Researchers and industry rely on registries to identify potential patients, understand the progression of a disease and the full range of symptoms, as well as help to plan clinical research. To achieve these goals, a registry must be a well-conceived, carefully planned effort. Join experts to learn about the planning process that is vital for developing a registry that has the greatest potential to help advance research.

       
      12:40 PM  -  1:45 PM
      Lunch
      Location: The Patio

      Sponsored by:

      Pfizer

      pfizer-logo square

      1:00 PM  -  2:00 PM
      Patient/Advocate Needs and Challenges Assessment
      Please provide your feedback to the attached live polls that will offer a better understanding of your community's current needs.
      2:00 PM  -  2:20 PM
      Closing Remarks
      Location: Grand Ballroom ABC
      Speakers:
       

      Track and Workshop Sessions - 2:30 PM

      2:30 PM  -  3:30 PM
      Track 1: Care & Support for Your Kids
      Location: Grand Ballroom D

      RARE Kids Growing Up: Experiencing Transitions in Care and Life

       

      As rare kids grow up, they encounter many periods of transition. Two of the most difficult transitions can be progressing from pediatric to clinical care, and transitioning into the workforce. Join experts who are working with the rare disease community to ensure that these transitions are successful for all and ultimately, a right fit for your loved ones, as they navigate into the next phase of their lives.

       

      Sponsored by:

      Horizon Pharma

      horizon-logo-square

      In Partnership With: 

      Child Neurology Foundation

      CNFXSm

       

       

      2:30 PM  -  3:30 PM
      Track 2: Legislative & Medical Advocacy
      Location: Grand Ballroom E

      Patient Advocacy: An Update on Federal Legislation

       

      Each year, Global Genes and its friends and partners work to bring you up to speed on the latest federal legislation impacting rare disease. Hear from advocacy experts on pending legislation affecting rare disease research, drug development, clinical care, efforts to advocate for change, and more, including what you can do to help.

      2:30 PM  -  3:30 PM
      Track 3: Drug Development: Getting into the Clinic
      Location: Grand Ballroom ABC

      Understanding the Constraints on Clinical Trials: Inclusion Criteria, Control Groups, Blinding, and More

       

      Drug development requires a series of clinical trials that test the new therapy. But these clinical trials must follow specific regulations and guidelines that are designed to ensure the ethical treatment of clinical trial participants and the safety and efficacy of the therapy. Join industry leaders to learn about the constraints placed on clinical trials, and have your questions answered about how the process works.

       

      Sponsored by:

      PRA Health Sciences

      pra

      Speakers:
      2:30 PM  -  3:30 PM
      Track 4: Science and Technology Innovation
      Location: Theatre

      Artificial Intelligence: Making Research and Diagnosis Faster and More Accurate

       

      Rare disease is often a race against time: time to diagnosis, time to conduct research, and time to develop therapies. It is compelling that with the advent of new science and technology such as artificial intelligence, diagnoses and treatments can be accelerated and more accurate. Learn how some leading innovators are applying new technologies to some of the greatest challenges that the rare disease community faces.

       

      Speakers:
      2:30 PM  -  3:30 PM
      Workshop 2: Development and Approval of New Therapies
      Location: Trabuco

      How to Talk with the FDA

       

      As patient advocates continue to become an integral part of the research and development process for therapies, discussions between advocates and the FDA are more becoming frequent and impactful. Understanding and preparing for the meeting is crucial to ensure that the advocate experience is influential in the regulatory process. Come hear two advocates share their experiences in talking with the FDA, and learn ways to increase the potential for achieving your goals.

       

      Track and Workshop Sessions - 3:40 PM

      3:40 PM  -  4:40 PM
      Track 2: Legislative & Medical Advocacy
      Location: Grand Ballroom E

      Advocacy for the Community: Newborn Screening

       

      Some of the biggest challenges faced by rare disease patients involve delayed diagnosis. Newborn screening can bring a name for the challenges facing a family, community support, and most importantly, the potential for better outcomes and quality-of-life when a treatment is available. Learn about this effort to expand newborn screening efforts through state legislation, and how your disease community can play a role.

      3:40 PM  -  4:40 PM
      Track 3: Drug Development: Getting into the Clinic
      Location: Grand Ballroom ABC

      Patients as Investors

       

      Patients are driving innovation and investment that is making an important difference in bringing therapies to market for rare disease. They can be more than partners in this process; patients can be investors and entrepreneurs. Learn about how, when, and where patients should be investing time, money, and other critical resources to help advance the pathway toward finding therapies and cures.

       

      Sponsored by:PRA Health Sciences

       

      Speakers:
      3:40 PM  -  4:40 PM
      Track 4: Science and Technology Innovation
      Location: Theatre

      Improving Quality of Life: 3D Printing Assistive Devices

       

      Advances in technology are improving the quality-of-life for rare disease patients. 3-D printing is one of the new technologies being used to help rare disease patients live a life with fewer limitations. Join patient advocates as they discuss their efforts to use 3-D printing to help improve patients lives.

      Speakers:
      3:40 PM  -  4:40 PM
      Workshop 1: Capacity Building for Your Organization
      Location: Saddleback

      Building Communities Together: The Benefits of the Global Genes Foundation Alliance

       

      Non-profit patient advocacy organizations can become part of a larger community through the Global Genes Foundation Alliance. The Alliance empowers advocates to share information and tips learned through their own experiences, and help others on the same path. But there are also other significant advantages of joining the Alliance, such as the ability to become part of an innovative, rare disease fundraising opportunity, in collaboration with the Festival of Children Foundation. Join us at this the session to learn more!

       
      5:30 PM  -  6:00 PM
      Cocktail Reception
      Location: Pre-Function Foyer

      Sponsored by:

      Vertex

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      6:00 PM  -  7:15 PM
      RARE Champion of Hope Awards
      Location: Grand Ballroom ABC
      Global Genes RARE Champion of Hope Awards provide an opportunity to recognize individuals who are true agents of change and innovation. Award nominees are honored for their commitment and dedication to the development of treatments, therapeutics, and levels of care that have improved the lives of patients and families who have been touched by rare disease.
      7:30 PM  -  9:45 PM
      Evening Reception and Party
      Location: The Patio
    • Saturday, 09/16/17
    •  
      12:00 PM  -  5:00 PM
      RARE Day of Beauty

      This event is exclusively for patients and advocates. You have the choice of a free nail, hair, or makeup appointment. All you have to do is choose what kind of treatment you want, an appointment time and then you’ll get pampered for free!

       
      Sign up for the RARE Day of Beauty event onsite at the RARE Patient Advocacy Summit on Friday, September 15th at registration from 7am-2pm. Spots are limited! 

       

      In Partnership With:

      Beauty Bus Foundation

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      Sponsored by:

      Horizon Pharma

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