2018 RARE Patient Advocacy Summit Agenda

Global Genes 2018 RARE Patient Advocacy Summit

Tuesday, October 2, 2018
11:30a – 5:00p

Child Neurology Foundation and Global Genes Peer Support Bootcamp

During the Peer Support Bootcamp, Child Neurology Foundation’s Family Support and Empowerment Program Peer Support Specialists will facilitate a discussion and share their real-life experiences in peer support and as parents of children with neurologic conditions. For more information and to apply, click here

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3:00p – 7:00pGeneral Registration Opens
SPONSORED BY  
3:30p – 5:30p

Freshman Orientation

The RARE Patient Advocacy Summit is filled with numerous opportunities for attendees to connect and learn. For first-time attendees, the breadth of opportunities can be overwhelming. This optional Freshman Orientation session will help first-time attendees get the most out of their Summit experience and navigate opportunities to best meet their personal interests. Attendees will hear from experienced Summit attendees, meet the Global Genes team, and receive technical support for the Summit app and partnering system. Attendees are also invited to join the Foundation Alliance Networking Reception following Orientation. Pre-registration required by emailing events@globalgenes.org.

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3:30p – 8:00pExhibits Open
Book signing available by Rachel Callander
5:30p – 6:00pMeet Your Advisor
6:00p – 8:00pFoundation Alliance Welcome Reception
SPONSORED BY  
8:00p – 9:00pDocumentary Viewing, “At The Edge of Hope”
SPONSORED BY  

 

Wednesday, October 3, 2018 – Day One
6:00a – 7:00aSunrise Yoga
6:30a – 5:30pGeneral Registration Opens
SPONSORED BY  
6:30a – 5:30pExhibits Open
Book signing available by Rachel Callander
6:30a – 8:00aNetworking Breakfast
SPONSORED BY  
8:15a – 9:15aWelcome and Keynote
SPONSORED BY  

Rising Above: Using Empowering Language in Health Care
Rachel Callander

When faced with a life-altering diagnosis, families find themselves drawing on their depths of resilience and their communities to understand, adapt and thrive. Healthcare professionals can be a tremendous resource for families, but all too often, the words healthcare professionals use as they try to help cause confusion, frustration, anger or hurt. With changes in language, families and medical professionals can realize understanding, reassurance and support. Inspired by her experiences as she learned about the diagnosis of her daughter Evie and as she loved and celebrated her, Rachel Callander engages with healthcare professionals to challenge the language used with regards to disability and the importance of effective and empowering communication in healthcare.

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9:30a – 10:30aMedical and Scientific Advisory Board (MSAB) Presents

Patients Driving Advances: From Research to Therapy

Unlike other stakeholders in the clinical research enterprise, patients and their families live and battle with disease on a daily basis. They have first-hand knowledge which can help to focus research on needed and valued questions and outcomes while strengthening participation in clinical research. Patients and families also share a sense of urgency, an understanding that a misstep or a delay in the research process can result in a heavy cost. Patients and researchers will share their stories on how patients can and should be powerful partners and drivers in research and therapy development.
Speaker: Luke Rosen, Founder, KIF1A.org, and Associate Director of Patient Engagement, Ovid Therapeutics
Speaker: Jeffrey Sherman, MD, FACP, Executive Vice President of Research and Development, and Chief Medical Officer, Horizon Pharmaceuticals
Speaker: Anthony Philippakis, PhD, MD, Chief Data Officer and Institute Scientist, Broad Institute of MIT and Harvard
Speaker: Daniel Fischer, Founder and Chief Executive Officer, Tevard Biosciences; and Program Director at the Massachusetts Institute of Technology (MIT); and Dravet Syndrome Parent Advocate

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10:30a – 2:00pPoster Presentations
10:30a – 11:15aNetworking Break
SPONSORED BY  
11:30a – 12:30pTrack Sessions
Day One

Track 1

Living with a Life-Altering Condition

(Searching for a Diagnosis -> Adapting to the New Norm -> Flourishing)
 
 
SPONSORED BY

Track 2

Architecting Your Disease Community

(Organizing -> Growing -> Sustaining and Adapting)
 
 
 
SPONSORED BY

Track 3

Patients as Partners and Drivers

(Discovering -> Targeting -> Building on Advances)
 
 
 
SPONSORED BY

Track 4

Science and Technology Innovation

(New Approaches for Discovery -> Building on Past Modalities -> New Approaches for Therapeutics)
 
SPONSORED BY

11:30a – 12:30p

Looking for Answers in Uncertainty: Managing When There is No Diagnosis or Prognosis

For many rare disease patients and their families, the journey begins with a struggle to obtain an accurate diagnosis and continues with an incomplete understanding of how the disease will progress. Managing the uncertainty that comes along with a missing or minimal diagnosis or with an unclear prognosis is a major challenge. This session will discuss strategies for managing forward through the unknown while making an important impact.
Speaker: Kimberly Bower, MD, Chief of the Division of Palliative Medicine, Rady’s Children’s Hospital, and Medical Director of the Pediatric Program, Scripps Hospice
Speaker: Caroline Loewy, MBA, Co-Founder and Board Member, KCNQ2 Cure Alliance
Speaker: Hillary Savoie, PhD, Founder and Co-Executive Director, The Cute Syndrome Foundation, and Writer

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Non-Profit 101: Launch and Governance of a Non-Profit

A non-profit organization can provide patients and their families with a community and a structured way of providing clinical and support resources, funding for research and a unified point of contact. But non-profits must adhere to a complex set of rules and regulations. Join leaders in non-profit management to learn how to create a non-profit, how to get a non-profit organization underway and how to successfully manage its internal operations.
Speaker: Dawn Ireland, President, CDH International
Speaker: Cristina Gomez, MPA, Assistant Vice President, CCS Fundraising

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Data, Data, Data: Sharing and Owning Data to Game Change Rare Disease Research

We are in the middle of a data revolution that promises to rapidly advance rare disease research. When data is gathered globally and multiple types of data are combined, opportunities to learn about and ultimately solve rare diseases are created. However, those opportunities are limited without ensuring data are open to study by all researchers. It is becoming more clear that sharing is often maximized when data is controlled by rare disease organizations, while creating responsible ways for doing so. Learn the advantages and practical strategies for collecting, sharing and maintaining data to advance rare disease research and treatment.
Speaker: Daniel MacArthur, PhD, Assistant Professor, Harvard Medical School and Massachusetts General Hospital, and Co-Director of Medical and Population Genetics, Broad Institute of MIT and Harvard and Center for Mendelian Genomics
Speaker: John Wilbanks, Chief Commons Officer, SAGE Bionetworks
Speaker: Jennifer Thornton, MSW, Executive Director, Children’s A-T Project

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Rare Research Resources: Animal Models and 3D-Engineered Tissues

Animal models and 3D-engineered tissues play a vital role in developing therapies for rare disease. Patient advocates need to understand how these technologies help provide a way to study rare diseases and test potential therapies for safety and efficacy. Different animal models have different advantages, and sometimes much can be learned from very simple models. However, animal models are not human tissues which can present challenges. Engineered tissues can help drive treatments because they can model the structure and function of human tissues and complement animal models.
Speaker: Ethan Perlstein, PhD, Chief Executive Officer, Perlara
Speaker: Danilo Tagle, MS, PhD, Associate Director for Special Initiatives, National Center for Advancing Translational Sciences (NCATS)
Speaker: Karen Erickson, Associate Executive Director of Community Engagement, Alpha-1 Foundation

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12:30p – 1:45pNetworking Lunch
SPONSORED BY  
2:00p – 3:00pTrack Sessions
2:00p – 3:00p

Setting Up Your Environment to Adapt to the New Normal

With a rare disease diagnosis comes the need to adapt — to find ways to align the needs and hopes of the patient and family with the reality of the symptoms, disease progression and available therapies. But a rare disease diagnosis does not have to equate with a limited life. Patients and families have found multiple ways to make adaptation an expansion of options rather than a limitation. Also, adaption does not always have to mean change. Incorporating things that were a part of daily life before the diagnosis is critical to try to keep the balance between stability and change. In this session, rare patients and family members will talk about how they found that balance and share strategies on how to adapt to the new opportunities and challenges.
Speaker: Kristin Smedley, President and Co-Founder, Curing Retinal Blindness Foundation
Speaker: Amy Price, Metachromatic Leukodystrophy Parent Advocate
Speaker: Perla Aparicio, CSW, Certified Social Worker, Champions Action Network

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Capacity Building Strategies: Managing Volunteers

Volunteers can be an invaluable resource for non-profit organizations, playing key roles in achieving program goals in addition to providing non-profits with a way to adapt rapidly to changing conditions and needs. Hear from leaders in the non-profit community about how to recruit volunteers, how to engage dedicated volunteers and how volunteer management is an organization-wide effort.
Speaker: Candie Davidson-Goldbronn, Associate Senior Vice President, Children’s Hospital of Los Angeles
Speaker: Deborah Fowler, President and Founder, Soft Bones

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Building Valuable Registries and Natural History Studies

One of the critical first steps in the research and drug development pathway is understanding how large a rare disease community is, where the patients are and what the progression of the disease looks like. These questions can be answered through patient registries and natural history studies. A robust, well-planned and detailed registry or natural history study can serve as a key resource for research partnerships and can even serve to facilitate clinical research. This session will cover the foundations of building these tools and how they can be expanded to advance research and drug development.
Speaker: Benjamin Forred, MBA, Translational Research Project Manager, Sanford Research
Speaker: Francis Kalush, MSc, PhD, Health Programs Coordinator for Professional Affairs and Stakeholder Engagement, Center for Drug Evaluation and Research (CDER), Food and Drug Administration
Speaker: Ashley Winslow, PhD, Senior Director of Translational Research and Portfolio Development, Orphan Disease Center, University of Pennsylvania
Speaker: Audrey Davidow Lapidus, Founder and President, Pitt Hopkins Research Foundation

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Rare Research Resources: CRISPR System and iPS Cells

CRISPR gene editing and stem cells show promise as potential therapies anticipated to have a major impact on treating rare diseases. These technologies are already making an impact as research tools used to uncover the underlying mechanisms of rare diseases and the testing of other potential therapies. CRISPR and induced pluripotent stem (iPS) cells can reduce the time and effort to conduct research on rare diseases by making it easier to develop animal models or by supplementing animal model research. This session will provide an overview of these approaches, focusing on the benefits of including CRISPR and iPS cell approaches in a rare disease research portfolio.
Speaker: Marcelo Coba, PhD, Assistant Professor of Psychiatry, Zilkha Neurogenetic Institute, Keck School of Medicine of University of Southern California
Speaker: Lisa Flint, Founder and Managing Director, The AADC Research Trust

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3:15p – 4:00p

Understanding the Emotional Health of Rare Disease Families

Rare disease affects the emotional health of the entire family, not just the patient. Family members may feel frustrated when they cannot relieve the physical and emotional stress of a loved one with the rare disease. Family members may also experience tremendous depression and anxiety due to grief over loss of what they thought their family life would be and relationship stress, including guilt and resentment due to the overwhelming burden of managing chronic disease in the home. Hear from leaders in counseling and rare disease advocacy about strategies and tools to find connections and celebrate a flourishing family life.
Speaker: Gay Grossman, Co-Founder, ADCY5.org, and Director of Patient Engagement, Neurogene
Speaker: Mousumi Bose, PhD, Scientific Advisory Board Committee Chair, Global Foundation for Peroxisomal Disorders, and Assistant Professor, Montclair State University
Speaker: Daniela Paolone, LMFT, Licensed Marriage Family Therapist, Westlake Village Conseling

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Sustaining an Organization: Managing Long-Term Growth and Development

It happens all too easily — an organization that began with a targeted mission can take on programs that may bring in donor funding but may not be targeted to their mission or meeting the needs of their community. An organization can also focus so much effort on fundraising and program development that planning for staff transitions is neglected. Join non-profit leaders to learn about ensuring the success and sustainability of your organization through detailed budgeting, program development and transition planning to ensure future growth and impact.
Speaker: Louise Vetter, President and Chief Executive Officer, Huntington’s Disease Society of America
Speaker: Lalé Welsh, Executive Director and Chief Executive Officer, Neuromuscular Disease Foundation (NDF)

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The Role of a Chief Science Officer: The Secret Sauce for Forward Movement in Research and Scientific Collaborations

Getting to a treatment or cure for rare diseases means engaging in scientific research. But you want to be sure that the scientific research moves toward a therapy rather than repeating old findings or pursuing an avenue of research with little promise. How can leaders of rare disease patient advocacy organizations assess the research landscape and accelerate research and drug development? One approach highlighted by many as a best practice is bringing on board a Chief Science Officer (CSO) to sort through the scientific information and provide dedicated, targeted guidance. Hear from rare disease leaders about how a CSO can transform your organization’s research efforts.
Speaker: Molly White, Chief Executive Officer, Myotonic Dystrophy Foundation
Speaker: John Porter, PhD, Neuromuscular Disease Consultant

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Rare Research Resources: Biomarkers

A biomarker can be used to diagnose or assess the progression of a disease by providing a way for someone other than the patient to objectively measure the medical signs of a disease. Biomarkers can be molecular, such as the amount of a protein, or physiological, such as stamina or muscular response. Accurate and easily-measured biomarkers are critical for rare disease drug development, allowing for assessment of treatment effects in clinical trials. This session provides an overview of where and how biomarkers are used in research and development in addition to how biomarkers are identified and validated.
Speaker: Stephen Maricich, PhD, MD, Medical Director, BioMarin Pharmaceutical, Inc
Speaker: Holly Kordasiewicz, PhD, Executive Director of the Neuroscience Drug Discovery and Biomarker Departments, Ionis Pharmaceuticals

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4:00p – 4:15pNetworking Break
SPONSORED BY  
4:15p – 5:00p

Update from the Community: Caregiver Survey

Caregivers are not often acknowledged when considering policy and social support measures for those affected by rare disease. A study of rare disease caregivers recently completed by the National Alliance for Caregiving in partnership with Global Genes demonstrates both the critical role and the unmet support needs of caregivers. Hear from leaders involved in this study about the key findings and policy solutions to ensure that the strengths of caregivers are included in rare disease research and treatment and that the needs of everyone affected by rare disease are met.
Speaker: C. Grace Whiting, JD, President and Chief Executive Officer, National Alliance for Caregiving
Speaker: Mousumi Bose, PhD, Scientific Advisory Board Committee Chair, Global Foundation for Peroxisomal Disorders, and Assistant Professor, Montclair State University

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Update from the Community: Patient-Focused Drug Development Meetings

Patients and caregivers have a unique and personal perspective on rare disease. For a long time, this valuable perspective was not a primary focus within research and drug development and regulatory review. From 2012 through 2017, the Food and Drug Administration (FDA) held 24 disease-specific Patient-Focused Drug Development (PFDD) meetings, including several for rare disease, to ensure patient perspectives are included in regulatory efforts. Patient organizations are now encouraged to organize meetings using the PFDD process as a model. In this session, leaders from patient organizations who are planning or who have conducted PFDD meetings will lead a discussion on the process, challenges and benefits of engaging in this effort to help ensure the understanding of the patients’ disease experience and their treatment priorities are included in drug development and review.
Speaker: Joy Aldrich, Charcot-Marie-Tooth (CMT) Advocacy Director, Hereditary Neuropathy Foundation
Speaker: Allison Moore, Founder and Chief Executive Officer, Hereditary Neuropathy Foundation (HNF)
Speaker: Jill Jarecki, PhD, Chief Scientific Officer, Cure SMA

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Changing Policy, Saving Lives

The rare disease community relies on state and federal legislation to help accelerate rare disease diagnosis, therapy development, clinical care and social support. Rare disease patients, families and caregivers can make a difference with policymakers, making sure that individual and community needs are considered in legislation. Join Global Genes’ legislative advocacy partners to hear updates on pending legislation and advocacy efforts that are critical for the community and learn the ins and outs of advocating for rare patients.
Speaker: Angela Ramirez Holmes, President, California Action Link for Rare Diseases (CAL Rare)
Speaker: Jennifer Bernstein, Executive Vice President, Horizon Government Affairs
Speaker: Julia Jenkins, Executive Director and Secretary of the Board of Directors, EveryLife Foundation for Rare Diseases
Speaker: Darlene Shelton, President and Founder, Danny’s Dose Alliance

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Update from the Community: Quality of Life Markers

Rare disease patients and caregivers have played a critical role in helping to elevate the importance of focusing on the whole patient when treating a rare disease. Treatments and clinical care must include efforts to address the physical symptoms of rare disease patient communities and researchers are increasingly realizing that addressing just physical symptoms of rare diseases is insufficient. Clinical care must also focus on improving quality-of-life for patients and caregivers — physically, emotionally and socially. But how do we measure these impacts? Join this session to learn more about cutting-edge research to improve how we measure quality-of-life and how we can include these measures in research on therapy development and clinical care practices.
Speaker: Maria Picone, Founder and Chief Executive Officer, TREND Community, and Prader-Willi Syndrome Parent Advocate
Speaker: Tiffany Westrich-Robertson, Chief Executive Officer, International Foundation for Autoimmune & Autoinflammatory Arthritis

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5:15p – 5:30p

THRIVE: Game Changing the World of Rare Disease through Collaboration

As members of the rare disease advocacy community, we understand that we are all an integral part of a much larger and growing ecosystem working to affect positive change in the rare disease space. Driving progress and development of treatments and therapies for rare disease is a team effort. Together we can do more than when we act alone. Several rare disease patient advocacy organizations have begun a movement to elevate collaborative values and commit to working together to improve the lives of so many. Join us as rare disease advocacy leaders launch the effort to THRIVE (Trust, Honest Communication, Respect, Intentional, Vulnerability, Engage and empower).
Speaker: Amy Miller, MA, MSN, PCNS-BC, Executive Director, Child Neurology Foundation

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5:30p – 6:30p

Lives Changed: Stories with Impact

We are here to celebrate our victories as a community — small and large — as we work each and every day to make lives better for our friends, families and loved ones impacted by rare disease. We are also here to celebrate the ways in which we lift each other up, the countless ways our stories support each other and the ways that sharing the small moments makes a big difference. Mike Porath will join us to celebrate the stories that are in us all and discuss the stories that link us together in wonder and in purpose.
Speaker: Mike Porath, Chief Executive Officer, The Mighty
Speaker: Gunnar Esiason, Program Director, Board Member, and Advocate, Boomer Esiason Foundation

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6:45p – 9:30pDay One Reception
SPONSORED BY   Shire

 

Thursday, October 4, 2018 – Day Two
6:00a – 7:00aSunrise Yoga
6:30a – 2:00pGeneral Registration Opens
SPONSORED BY  
6:30a – 2:00pExhibits Open
Book signing available by Rachel Callander
6:30a – 8:00aNetworking Breakfast
SPONSORED BY  
8:15a – 9:30aWelcome and Main Session

Rare Disease Updates from the Global Stage

Rare disease unites patients and families across borders. The goals to accelerate research to understand rare diseases; ensure patients receive accurate, rapid diagnoses; identify and support best standards for clinical care; and support efforts to develop effective therapies and cures are universal. Hear from global advocacy leaders about ambitious, innovative projects aimed at achieving all of these goals including the International Rare Disease Research Consortium (IRDiRC), The Global Commission to End the Diagnostic Odyssey for Children with a Rare Disease and Global Alliance for Genomics and Health.
Speaker: Anthony Philippakis, MD, PhD, Chief Data Officer and Institute Scientist, Broad Institute of MIT and Harvard
Speaker: Steve Groft, Senior Advisor to Director, National Center for Advancing Translational Sciences (NCATS), NIH
Speaker: Clifford Goldsmith, MD, Managing Director for US Providers, Microsoft
Speaker: Barbara Wuebbels, MS, RN, Chairman, Professional Patient Advocates in Life Sciences
Speaker: Frank Sasinowski, MS, MPH, JD, Director, Hyman, Phelps & McNamara, PC
Speaker: Robin Smith, MBA, MD, President and Chairman of the Board, Cura Foundation

9:45a – 3:00pTrack Sessions
9:30a – 4:00p

Entrepreneurial Readiness Bootcamp

Overview:
The Entrepreneurial Readiness Bootcamp is designed for patient advocates who fund rare disease research and are looking to better coordinate and build structure around their efforts. Space is limited for this hands-on and in-depth day of learning, so anyone interested in attending must apply and participate for the full day.
If you can answer yes to these questions, we encourage you to apply:

  1. Are you actively funding rare disease research?
  2. Are you currently or do you have a plan to take the research to therapeutic
    discovery/development?
  3. Are you considering creating a company?

Apply Now

Day Two

Track 1

Living with a Life-Altering Condition

(Diagnosis -> Adapting to the New Norm -> Flourishing)
 
 
SPONSORED BY

Track 2

Architecting Your Disease Community

(Organizing -> Growing -> Sustaining and Adapting)
 
 
SPONSORED BY

Track 3

Patients as Partners and Drivers
(Discovering -> Targeting -> Building on Advances)
 
 
 
SPONSORED BY

Track 4

Science and Technology Innovation
(Building on Past Modalities -> New Approaches for Discovery -> New Approaches for Therapeutics
 
SPONSORED BY

Track 5

Entrepr-
eneurial Readiness Bootcamp

Invitation only

Are you a patient advocate who is funding rare disease research and looking to build structure around your efforts? Apply to take part in this hands-on, in-depth, full day program.

Apply Now

9:45 – 10:45

Understanding the Emotional Health of Rare Disease Patients

Rare disease patients may find themselves experiencing not just the physical challenges of the disease, but also grief for lost opportunities and expectations, anxiety over what is known and unknown about the rare disease, and depression. Join us for a discussion of these challenges shared throughout the rare disease patient community and what support resources and tools are available to help you thrive.
Speaker: Allison Fine, MSW, LICSW, Founder and Executive Director, Center for Chronic Illness
Speaker: Seth Rotberg, Board Member, Huntington’s Disease Youth Organization

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Organizing and Mobilizing Your Community of Stakeholders Regionally and Globally

Rare disease patients and families are a community united through the experience of the disease. To support patients and families, to strengthen the clinical care and to develop treatments to improve their quality of life, the patient community must be organized and mobilized to work together. This requires developing strong connections, building trust and uniting. Hear from two patient advocates who are working to develop and implement community action roadmaps to ensure a better future for patients and families.
Speaker: Mary Beth Campbell, Director of Corporate Partnerships, California Institute of Technology, and Bloom Syndrome Parent Advocate
Speaker: William Davis, Chief Executive Officer, A-T Society

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We Can’t Do It Alone: Structure and Governance for Research Collaborations

Collaboration is key to advance research for rare disease. Collaborations may be small, between one researcher and one advocacy organization with a individual research agreement, or more complicated, involving several organizations that address a shared need with a formal governance structure. There are also many forms of collaboration in-between involving a few to several partners. All successful collaborations involve trust, transparency and flexibility. Leaders from several organizations will discuss the structure, development and maintenance of research collaborations and how to ensure strong collaborations for advancing rare disease.
Speaker: Hans Schlecht, MD, Attending Physician of Internal Medicine and Infectious Disease, Baystate Health, and SYNGAP1 Parent Advocate
Speaker: Douglas Macdonald, PhD, Director of Research Operations and Scientific Alliances, CHDI Management / CHDI Foundation
Speaker: Michelle Davis, Executive Director, International Fibrodysplasia Ossificans Progressiva (FOP) Association

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Rare Therapies: Pharmacological Chaperones and Cell Therapies

Most rare diseases are caused by genetic mutations. Often, the mutations result in a protein that does not have the shape necessary to successfully perform its function. New approaches are being developed to address these causes of rare disease through cell therapies or pharmacological chaperones which help proteins take on the shape needed to get to where they need to be within cells. Modified cells, or cells combined with other molecules, may also correct the genetic problem in tissues, or replace tissues entirely. Join us for a discussion of these new approaches and how they may be used as rare disease therapies.
Speaker: Shaun Lee, PhD, Monahan Family Associate Professor of Rare and Neglected Diseases, University of Notre Dame
Speaker: Weston Miller, MD, Medical Director, Sangamo Therapeutics

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9:30a – 12:30p
Welcome

Speaker: Walter Kowtoniuk, PhD, Principal, Third Rock Ventures
Speaker: Yael Weiss, PhD, MD, Vice President of Business Development, Ultragenyx Pharmaceuticals
Speaker: Steven Laffoon, Chairman, President, and Co-Founder, Wylder Nation

Why Start a Company, and When is the Time Right?

Starting a company is a significant undertaking, and the time must be right to ensure the greatest chance for success. The morning sessions will focus on understanding when starting a company is a viable option for developing a rare disease treatment through discussions on the aspects of rare disease that present challenges for companies, when the science has reached a point for translation into therapy development, what kind of plan is needed to maximize the strength of a company and how to be sure the company has sufficient financial support.

● Unique Opportunities and Challenges in Rare Disease
Speaker: Jeremy Levin, DPhil, MB BChir, Chairman and CEO, Ovid Therapeutics, Inc.
● How Do you Know When the Science is Ready?
Speaker: Daniel Fischer, Founder and Chief Executive Officer, Tevard Biosciences; and Program Director at the Massachusetts Institute of Technology (MIT); and Dravet Syndrome Parent Advocate
● Developing a Multi-year Plan: Milestones and Value Creation
Speaker: Walter Kowtoniuk, PhD, Principal, Third Rock Ventures
Speaker: Heather Behanna, PhD, Principal, Sofinnova Ventures
● Capital: How to Raise Money Over Time
Speaker: Neil Kumar, PhD, Chief Executive Officer, BridgeBio LLC

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1:30p – 4:00p Making It Happen: Tactical Modules

Starting a company requires consideration of a lot of details. How do you want to structure the research and development, financial, and management teams? How does partnering with other companies play a role in the development of your company? What legal steps and corporate governance do you need? The afternoon session will focus on these questions from the perspective of founding a company through the efforts to grow and expand a company.

● Building the Team

  • Virtual Versus Brick and Mortar
  • In-House Versus Externalizing
  • Consultants, Founders, and the Scientific Advisory Board
    Speaker: Andrea DiMella, Recruiting Director, Third Rock Ventures
    Speaker: Megan Baierlein, Vice President of Program Management, Audentes Therapeutics
  • ● The Role of Partnering with Biopharmaceutical Companies
    Speaker: Yael Weiss, PhD, MD, Vice President of Business Development, Ultragenyx Pharmaceuticals
    Speaker: Fran Sverdrup, PhD, Associate Professor of Biochemistry and Molecular Biology, Saint Louis University School of Medicine

    ● Corporate and Legal Fundamentals: Incorporating, Ownership and Intellectual Property
    Speaker: Christina MacDougall, JD, PhD, Associate, Morgan, Lewis & Bockius LLP

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10:45a – 2:00pPoster Presentations
10:45a – 11:30aNetworking Break
SPONSORED BY  
11:30a – 12:30pTrack Sessions
11:30 – 12:30

Modern Therapies for Emotional Health: Different Ways to Cope

When managing a rare disease, easing the symptoms of the disease itself are only one part of a therapy. Strengthening the emotional health and quality of life for both patients and families is often equally important. Recently, there has been a deepened understanding of the value of complementary therapies aimed at helping patients and families cope with symptoms or the effects on emotional health of rare diseases. There are many different types of complementary therapies such as yoga, meditation, massage therapy, expressive therapies such as art or dance or music and recreational therapies such as horseback riding, swimming or other adventure programs. This session will provide an overview of these modern therapy approaches, how they may help and resources for trying them.
Speaker: Laura Fonseca, MT-BC, Clinical Director and Music Therapist, Meli Musi
Speaker: Lucy Wong, Sclerodoma Patient Advocate and Artist
Speaker: Jon Miller, Co-Founder and President, Network of Tyrosinemia Advocates
Speaker: Amanda Miller, Co-Founder and Board member, Network of Tyrosinemia Advocates

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Clinical Care: Building Standards of Care for Your Disease Community

Rare disease patients often find themselves with very little information or a roadmap of care for their disease. Clinical standards of care can make a tremendous difference for a rare disease community by establishing quality standards for diagnosis, treatment and monitoring. Rare disease patients often must become architects of their disease, working with clinicians to help design comprehensive care standards. Join these real world experts to learn about how to develop standards of care that integrate the perspectives of patients, scientists and medical professionals.
Speaker: Kathi Kinnett, MSN, APRN, Senior Vice President of Clinical Care, Parent Project Muscular Dystrophy
Speaker: Jeffrey Buchhalter, PhD, MD, Adjunct Professor of Pediatrics, University of Calgary

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Is the Research Plan Working? Evaluation and Accountability for Research Grants and Contracts

Rare patient advocacy organizations are increasingly taking a lead in funding and focusing discovery on clinical research. With this responsibility comes the need for a strong research plan with required milestone assessments of progress. How should your organization structure its evaluation of the research plan? What data are important to gather from researchers? How should you measure success? How do you use this evaluation to build on your successes? This session will look at the answers to these questions and more.
Speaker: Tracy Dixon-Salazar, PhD, Director of Research and Strategy, LGS Foundation
Speaker:Karen Erickson, Associate Executive Director of Community Engagement, Alpha-1 Foundation

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Rare Therapies: A New Era for Genetic Medicines

New types of therapies, such as gene therapy and oligonucleotides, are paving the way toward treating rare diseases that have so far been thought of as “undruggable”. Gene therapy focuses on adding a corrected copy of a gene or directly altering a mutated gene. Oligonucleotide therapies focus on blocking the formation of a protein or restoring the function of a protein. In this session, leaders from patient advocacy and industry will discuss the promise of these new approaches.
Speaker: Bartholomew Tortella, MBA, MD, Global Medical Affairs Lead of Rare Disease Hematology and Transplantation, Pfizer
Speaker: Alex Mori

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12:30p – 1:45pNetworking Lunch
SPONSORED BY  
2:00 – 3:00

Relax and Refocus: Emotional Health Therapies

Modern therapies encompass the emotional as well as the physical health of rare disease patients and families. These approaches aim to improve the effect of the rare disease in addition to helping patients and families cope with the emotional challenges they face. Join experts in this experiential session that will introduce you to music therapy and guided meditation as tools to relax and refocus.
Speaker: Anushe Shoro, Special Projects Coordinator, CCS Fundraising
Speaker: Laura Fonseca, MT-BC, Clinical Director and Music Therapist, Meli Music
Speaker: Tom Chester, Program Director, Sing Me A Story Foundation
Speaker: Austin Atteberry, MBA, Founder and Executive Directory, Sing Me A Story Foundation

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Patient Advocacy In and Out of Industry: What You Need to Know About Working Together

The importance of collaborations between patients and industry is obvious in that no treatment or cure will ever be developed without a strong partnership. In order to collaborate effectively, knowledge about the differences of working with industry versus working in industry is critical. Understanding the priorities and motivation for each stakeholder is critical as they may differ. This session will discuss those different advocacy roles, how to identify overlapping goals between industry and rare disease, and how to measure the effectiveness of these collaborations.
Speaker: Alan Gilstrap, Executive Director of Advocacy and Policy, Akcea
Speaker: Sandra Revill Tremulis, Founder and President, The Lipoprotein(a) Foundation
Speaker: Jenna Swan, Manager of Patient Advocacy, Retrophin
Speaker: Chelsey Fix, MPH, Associate Director of Industry and Government Relations, NephCure Kidney International

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The Nuts and Bolts of Collaborations: Prepare for the Paperwork

The Nuts and Bolts of Collaborations: Prepare for the Paperwork
All strong collaborations are built on trust as well as clear expectations and written agreements. Collaborations often require the same types of written documents, such as research agreements, confidentiality agreements, and material transfer agreements. Hear from organizational leaders about the needed components for collaboration agreements and how to develop templates to make the collaboration negotiation process more effective and efficient.
Speaker: Ami Gadhia, JD, LLM. CLP, Technology Transfer and Patenting Specialist of the Office of Strategic Alliances, National Center for Advancing Translational Sciences (NCATS)
Speaker: Mary Beth Campbell, Director of Corporate Partnerships, California Institute of Technology, and Bloom Syndrome Parent Advocate

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Rare Therapies: Monoclonal Antibodies and Enzyme Replacement

Potential rare disease treatments often focus on replacing what is missing. Enzyme replacement therapy focuses on providing a missing enzyme while monoclonal antibody therapy focuses on helping the immune system recognize cells causing damage that it typically does not target. This session will include discussions on how enzyme replacement and monoclonal antibody therapies work and discuss future possibilities for these therapeutic approaches in developing rare disease treatments and cures.
Speaker: Andrew Baik, PhD, Scientist, Regeneron
Speaker: Ekaterina Wright, MBA, MD, PhD, Global Medical Lead, Shire

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3:00p – 4:00p

Closing Session – Data and Technological Innovation

New technological innovations are revolutionizing the type and amount of data that can be collected when studying a rare disease. This, in turn, strengthens understanding of the progression of a rare disease and/or the effectiveness of potential treatments. These innovations provide a way not just to collect better data, but also to collect data that is truly patient-centric. Hear from industry and patient advocacy leaders about the exciting applications of these new tools.
Speaker: Scott Schliebner, MPH, Senior Vice President of Center for Rare Diseases, PRA Health Sciences
Speaker: Allison Moore, Founder and Chief Executive Officer, Hereditary Neuropathy Foundation (HNF)

4:00p – 5:00pBreak
5:00p – 6:00p

6:15p – 10:00p

RARE Champions of Hope Reception

RARE Champions of Hope Celebration
SPONSORED BY   PRa Health sciences

Join us for the RARE Champions of Hope Awards presentation, entertainment, and dinner.

 

Friday, October 5, 2018
8:00a – 12:00pRare Access to Critical Therapies (ACT) Stakeholder Summit
11:30a – 4:00p

Global Advocacy Leadership Symposium (attendance is now full)

Global Genes envisions a strong, collaborative and vibrant rare disease community across the globe – whereby all regions are equipped to address the growing challenges of rare disease. The Symposium will bring together global advocacy leaders to identify regional needs, problem-solve solutions, and share best practices to build strong rare disease communities in all regions globally.

** All participants must complete the needs assessment in advance. For more information please send inquiries to events@globalgenes.org with the subject line, “RARE Global Advocacy Leadership Symposium Inquiry”.