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2/28/2020 9:30:00 AM2/28/2020 2:00:00 PM 2020 Rare Disease Day The last day of February has been designated as Rare Disease Day in Massachusetts to call attention to the public health issues associated with rare diseases, which affect nearly 30 million Americans and countless others around the world. The event recognizes Rare Disease Day and the research being done in Massachusetts to treat and cure rare disease.
This year’s agenda will feature two panels: one from the patient perspective on how they’ve turned their diagnoses into their life’s work, finding empowerment through advocacy and action; and one from the industry’s perspective on how researchers and providers are not only tailoring a therapy for a specific patient but also the entire patient experience, and how this outlook has shaped their careers.
Alone we are rare, together we are strong.
To inquire about sponsorship opportunities, contact Laura Rudberg. Omni Parker House, 60 School St, Boston, MA 02108
Patient Advocate, LGMD2B, Muscular Dystrophy Association
Chris Anselmo, 33, currently serves as a market intelligence manager on the Healthcare Partnerships team within the Muscular Dystrophy Association’s national office. Chris has a personal connection to MDA’s mission — he is also an individual living with Miyoshi myopathy/limb-girdle muscular dystrophy type 2B (LGMD2B), an adult-onset form of muscular dystrophy. For the first 21 years of his life, Chris had no signs of muscle weakness. Then one night in 2008, while going for a run, he noticed his legs tired out quickly. Over the next 12 years, Chris has experienced progressive muscle weakness, numerous falls, and a gradual loss of mobility.
Today, Chris is a patient advocate who is passionate about raising awareness for rare diseases, educating healthcare stakeholders on the power of the patient experience, and helping anyone dealing with adversity — especially newly diagnosed patients navigating the ups and downs of a life-changing diagnosis.
Chris holds an MBA from Boston College and a bachelor’s degree in marketing from Northeastern University.
Director of Caregiver Support Services and Public Relations Frontotemporal Disorders Unit, MGH
Katie Brandt, MM is the Director of Caregiver Support Services and Public Relations in the Frontotemporal Disorders (FTD) Unit at Massachusetts General Hospital. Through this role, she provides leadership for research, education and community events aimed at understanding and supporting the caregiver experience for families impacted by Alzheimer’s Disease, Frontotemporal Disorders, atypical and young-onset dementias. As Co-Chair of the National Alzheimer’s Project Act (NAPA) Advisory Council on Alzheimer’s Research, Care and Services, Katie is able to connect with experts in the field of Alzheimer’s Disease and related dementias and contribute to federal policy initiatives and research priorities that promote health equity and person-centered care at every stage of dementia. Katie is a passionate advocate in the rare disease community, inspired by her personal experience as a former FTD caregiver for her late husband, Michael. Today, Katie is an Alzheimer’s caregiver for her father, Tom. Her experiences of love and loss embolden her to keep moving forward, determined that the cure of tomorrow is not so far from the care of today.
As President and CEO of the Massachusetts Biotechnology Council, Bob’s mission is to advance Massachusetts' leadership in the life sciences to grow the industry, add value to the healthcare system and improve patient lives. Over the last decade, Bob has truly become a champion for patients by ensuring innovative companies have the best environment possible to research, develop, and commercialize breakthrough therapies and cures for people around the world who need and deserve them.
Bob has spent his career in both the public and private sectors. Before joining MassBio, he served as the Undersecretary of Economic Development within Governor Deval Patrick's administration, where he prioritized both healthcare and economic development issues and was a strong advocate for the life sciences industry in Massachusetts. Prior to that, he was elected as State Representative to the 11th Norfolk district for three terms. Bob has also held senior executive positions in the environmental services, capital management and venture capital industries. He brings passion and energy to all of his endeavors.
As Chief Experience Officer at Cambridge BioMarketing, Annemarie designs personalized omnichannel experiences for healthcare providers and patients with rare diseases. She has been instrumental in a number of rare disease product launches and led the development of CB’s predictive analytics and precision targeting platform. Using this proprietary approach, she helps clients find their target HCPs and develop individualized campaigns to reach them. She brings empathy and a strong passion for patients into everything she does, which led to her role in producing the Rare in Common documentary and podcast.
Robert J. Gould joined Fulcrum as president and CEO at the time of the company’s launch in 2016, bringing over 30 years of experience to the role. Previously, he served as president and CEO of Epizyme from 2010 to 2015. Prior to joining Epizyme, Robert served as director of novel therapeutics at Broad Institute of MIT and Harvard from 2006 to 2010. Robert spent 23 years at Merck where he held a variety of leadership positions, culminating in the role of vice president, licensing and external research. During his time at Merck, Robert was instrumental in advancing more than 20 compounds from discovery into clinical development in multiple therapeutic areas. Robert received a bachelor’s from Spring Arbor University and a Ph.D. from the University of Iowa and completed postdoctoral studies at Johns Hopkins University.
Kate brings an impressive breadth of experience and leadership within the biopharmaceutical industry, as well as a substantial background in business development, commercial and strategic planning, and program management. Kate previously served as Chief Business Officer of Blueprint Medicines from January 2016 to January 2019. Prior to joining Blueprint Medicines, she served as Vice President, Rare Diseases and Oncology Program Leadership at Idera Pharmaceuticals. In this role, she oversaw all aspects of the product development strategy for Idera Pharmaceuticals’ rare disease and oncology pipeline programs, including preclinical research, manufacturing and drug supply, regulatory affairs, clinical development and execution and commercial planning. Prior to joining Idera Pharmaceuticals, Kate was Head of Commercial Development at Sarepta Therapeutics, where she was responsible for product development and commercial planning and for cultivating relationships with key opinion leaders and patient advocacy groups. In addition, Kate previously served as Executive Director of Commercial Development at PTC Therapeutics and held corporate development and project management roles at Genzyme. She holds a B.A. from Wesleyan University with a double major in Biochemistry/Molecular Biology and Economics and an M.B.A. from Harvard Business School.
Hannah Kane (R – Shrewsbury) serves as the State Representative for the Eleventh Worcester District, representing the towns of Shrewsbury and Westborough (precincts 4 and 5), and was sworn in to serve her third term in January of 2019.
Hannah graduated from Boston University’s School of Management in 1993. Hannah has significant experience in both the public and private sectors. Hannah serves as a Member of the Joint Committee on Ways and Means, the Ranking Minority Member of the Joint Committee on Public Health, and the Ranking Minority Member of the Joint Committee on Cannabis Policy. Hannah is a member of the Central MA Opioid Task Force, a State Director for the national Women in Government Foundation, and a Board Member of the Massachusetts Caucus of Women Legislators. Hannah serves as Minority Leader Bradley H. Jones’ designee on the Massachusetts Food Policy Council and the Massachusetts Commission on Unaccompanied Homeless Youth and was appointed by Governor Baker to the Commission on Family Care and Child Care Services. She served on the Special Commission on Local and Regional Public Health and on the 2018-2019 Regional Transit Authority Task Force. She is a Founder and Co-chair of the first in the nation Food System Caucus. Hannah received a 2019 Massachusetts Public Health Association Legislative Leadership Award in recognition of her commitment to building an effective, efficient, and equitable local public health system. She also received a 2017 American Nursing Association of Massachusetts Friend of Nursing Award for her work on marijuana policy.
Hannah and her husband Jim live in Shrewsbury with their three children, one of whom has two chronic diseases.
SVP, Translational Medicine Mitobridge, An Astellas Co.
George has 20 years of experience in drug discovery, translational research and clinical drug development. Prior to the 2018 acquisition by Astellas, George was Vice President of Translational Medicine at Mitobrige, Inc., and earlier was Senior Director of Translational Medicine at Takeda Pharmaceuticals. George led translational research for multiple programs that target the ubiquitin-proteasome system, including several first-in-class molecules that are now approved or in clinical development. The research spanned both model systems and clinical trials, including mechanism of action and patient selection. While at Takeda and earlier within Millennium Pharmaceuticals, George contributed to clinical research and targeted development of the proteasome inhibitors bortezomib (VELCADE) and ixazomib (NINLARO). In this role he led the clinical pharmacogenomics research to define mechanisms of sensitivity and resistance to proteasome inhibition in different cancers and also collaborated with academic centers, pharmaceutical partners and patient advocacy leaders to initiate a broad personalized medicine initiative in myeloma. Prior to Millennium Pharmaceuticals, George led personalized medicine strategies at the start-up Millennium Predictive Medicine. George received his BS degree in Biology from Fordham University and his Ph.D. in Cellular Biology from SUNY Stony Brook. After thesis research at the Cold Spring Harbor Laboratory in New York he completed a postdoctoral fellowship at the MIT Center for Cancer Research.
Lexi Pappas is an advocate for and is also diagnosed with FSHD, a type of Muscular Dystrophy. When she was first diagnosed she would hide her symptoms from friends and classmates, but as things progressed, she decided to open up and start spreading awareness about it. In 2016 she released a documentary sharing how her family lives with FSHD daily, and the struggles that come along with it. The documentary now has over 45,000 views on YouTube and was featured on Fox news Boston and many other sites. Since then, she has spoken in front of the FDA and NIH, collaborated with multiple research companies, created YouTube videos, held fundraisers, and made relationships with others living with FSHD along the way. When we are all in a room together, it no longer feels like a rare disease and that is what makes those connections so special. In daily life, Lexi is a video editor and producer at Dell Technologies, and volunteers her time editing videos for non-profit organizations.
Dr. Ragan is a founder, as well as the Chief Executive Officer and President of X4. She has more than 19 years of experience building companies in the biotechnology industry. Most recently, Dr. Ragan consulted as Chief Business Officer at Lysosomal Therapeutics Inc (LTI), an Atlas Venture portfolio company, where she led the company’s business development activities. Prior to LTI, Dr. Ragan held leadership roles in corporate development and operations at Genzyme/Sanofi where she led strategic partnering efforts for Genzyme’s Rare Disease business and headed the supply chain planning for Genzyme’s flagship commercial products. Other professional roles include business roles at Hydra Biosciences, Oscient Corporation and Celera Corporation. Dr. Ragan received her BS in Mechanical Engineering from Tufts University and her PhD from MIT. She completed post-doctoral studies at Harvard Medical School.
Seth is a patient advocate and motivational speaker who is passionate about bringing his personal experience to better support the health community. He has over 10 years of fundraising, advocacy, and volunteer experience within the health space. His passion is driven by his mother’s 17-year battle with the rare, genetic disease known as Huntington’s Disease (HD). At the age of 20, Seth also tested positive for the disease and is a gene carrier. Rather than dwelling on these results, he decided to use them as motivation to fight back and get more involved within the community.
In 2019 he co-founded the nonprofit, Our Odyssey, to connect young adults in the rare and chronic communities with social and emotional support. In addition, he also sits on the Board of Trustees for the Huntington’s Disease Youth Organization (HDYO) and works professionally as the Patient Engagement Manager for Inspire.
Head, Hematology and Rare Disease, Takeda Pharmaceuticals
Cheryl joined Takeda in September 2019 as the Head of the Rare Disease U.S. Business Unit. At Takeda, she works to build on the strength of Takeda’s rare disease portfolio and capabilities focusing on rare bleeding disorders, lysosomal storage disorders (LSD), hereditary angioedema (HAE) and endocrine treatments.
As a global, values-based, science-driven biopharmaceutical leader, Takeda aspires to transform the treatment of rare diseases and is committed to helping enable faster diagnosis of rare diseases and access to treatments. This commitment includes spearheading initiatives like the Global Commission to End the Diagnostic Odyssey for Children with a Rare Disease, a multidisciplinary group of experts from around the world who have brought their creativity, technological expertise, and passion to accelerate the time to diagnosis.
With a strong legacy of treating rare diseases, Takeda puts the patient at the center of everything they do by focusing on the core Takeda-ism pillars of patient, trust, reputation and business (PTRB) to guide their rare disease efforts. This year, Takeda is celebrating Rare Disease Day by honoring a "Constellation of Rare Stars," and sharing the stories of 29 patients, families, caregivers, health care professionals and community members who have helped to “Reframe Rare.”
Shauna Sperou is 15 years old and was born with a rare genetic disease called Rothmund-Thomson Syndrome. She is a Freshman in High School and has been enjoying her time on a Robotics team for the past 3 years. She is very involved with RTS Foundation, advocating about her disease and raising money for research. She has also been involved in NORD's Running For Rare community for the past 6 years.