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. Alone we are rare, together we are strong. Our agenda feature two panels with different points of view of the issue: a patient panel, which will discuss the struggles beyond diagnosis and treatment; and an industry panel, which will focus on how the biotech and pharma industry is going beyond treatment to healing the whole patient, as well as supporting the caregivers.
Director of Caregiver Support Services and Public Relations Frontotemporal Disorders Unit, MGH
Katie became connected to the rare disease community when her husband was diagnosed with the rare neurological condition Frontotemporal Dementia at the age of 29. After his passing in 2012, she became a powerful public speaker, advocate, educator and trainer in the areas of caregiver support, Frontotemporal Degeneration (FTD) and the impact of dementia on caregivers and families. Katie has presented at colleges, conferences, hospitals, the Massachusetts State House and on Capitol Hill to raise awareness for young-onset dementia. Katie is the Director of Caregiver Support Services and Public Relations for the Massachusetts General Hospital Frontotemporal Disorders Unit and a member of the federal National Alzheimer’s Project Act (NAPA) Advisory Council on Alzheimer’s Research, Care and Services. She is a lead volunteer for the Association for Frontotemporal Degeneration and a co-facilitator of the Boston-area FTD Support Group.
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.
Lisa is a seasoned advocate, motivational speaker and Co-Founder of Sisters@Heart Foundation. a nonprofit whose mission is to improve the lives of those affected by heart disease and stroke through funding research projects and providing financial assistance to heart and stroke families in need. She is also involved as a Committee Member in Rare New England, a nonprofit dedicated to spreading awareness and providing education to stakeholders in the rare disease community.
With her first being 20 years ago, Lisa has survived four strokes and was finally correctly diagnosed with Moyamoya Disease after her last stroke four years ago. Lisa underwent double brain surgery to treat her rare disease far from home to restore her health. For years, Lisa has engaged as an advocate, lobbyist and volunteer with the American Heart Association and has served as a Go Red for Women National Spokesperson. Today, Lisa speaks all over the country as a motivational survivor, patient advocate and stroke and heart health ambassador with hundreds of media engagements under her belt. Lisa lives in Massachusetts with her husband and two children. To learn more about Lisa, visit lisadeck.com or follow her on Facebook, Twitter and Instagram.
Associate Director, Global Operations and Patient Advocacy, Aegerion Pharmaceuticals
Marta has 20 years’ experience in the biotech industry. She has been with Aegerion Pharmaceuticals in Cambridge MA since 2013. She has broad experience ranging from commercial operations, supply chain, and patient advocacy. Most recently, Marta has worked to facilitate importation of drug products through Named Patient Access and has built strong partnerships with patient communities in the US and International markets, focusing on rare disease products. Previously she was with Genzyme, working in the International Charitable Access Program which provided access to treatments for patients worldwide.
Marta holds a Bachelor of Science in Business Management and is currently a candidate for a Master of Science in Project Management and Operations (Jan 2020).
Muscular Dystrophy Massachusetts State Ambassador, and business owner of Girls Chronically Rock
Keisha Greaves is a motivational speaker, the founder of Girls Chronically Rock, and the Massachusetts State Ambassador for the Muscular Dystrophy Association (MDA). Girls Chronically Rock (www.girlschronicallyrock.com) offers inspired fashion celebrating Muscular Dystrophy and other chronic illnesses. Over the past few years, Keisha has been featured in Improper Bostonian, Boston Voyager, Herself 360, Liz on Biz, among other outlets on and offline. She’s lent her voice to audiences at the Spaulding Rehabilitation Center in Charlestown, Massachusetts General Hospital, the Boston Muscle Walk, local “Fill the Boot” events, Cambridge City Hall, and, most recently, Girl’s Night Out for Muscular Dystrophy – a Boston-area fundraising event held by NextonScene.
Mariah is the Patient Advocacy Director at Pfizer’s Rare Disease Research Unit in Cambridge, MA. Mariah’s 15 years of extensive experience leading innovative collaborations with advocacy associations and healthcare organizations has positively impacted the lives of patients and the broader Rare Disease community. She passionately ensures patient perspectives and insights are gathered and incorporated into the earliest stages of Rare Disease drug development to advance access to novel therapies. She has also held numerous commercial positions in the Alzheimer’s disease, cardiovascular, multiple sclerosis and psychiatric franchises across research, marketing and sales management. Mariah received a Bachelor of Science in Nursing form Worcester State College.
Duchenne’s Muscular Dystrophy Patient and advocate
Austin is a 20 year old sophomore , currently working on a mechanical engineering degree at Bunker Hill Community College. In his free time, he enjoys playing power soccer with his team in weekly practice and in games around the United states, and downhill skiing with his family. Austin has been a participant in clinical trials, is a volunteer on Parent Project Muscular Dystrophy’s Adult Patient Advisory Committee, and a member of the review board for certified duchenne care centers. Whenever possible, he uses his voice to elevate our DMD community and increase awareness of the need for approved drug therapies. Austin spoke at the first ever advisory committee meeting for a duchenne drug approval, has testified at congressional briefings and met directly with the FDA representing the duchenne community. As the subject of documentaries, including, To The Edge Of The Sky, Rare In Common, and Gene Doctors, and the recipient of the 2016 Meyer- Whaley instrument of change award , Austin has been committed to using his life situation to raise awareness for those living with rare disease. Practically, he would like to be involved in technology that can make the lives of those affected easier, and help to measure benefit in clinical trial setting so more non-ambulant young men will be able to participate and give valuable data.
Executive Vice President, R&D and Chief Medical Officer, Sarepta
In 2003, Dr. O’Neill joined Biogen as an associate director of clinical development, making the transition from practicing neurology full-time at Massachusetts General Hospital (MGH) to industry. During his 15-year career with Biogen, Dr. O’Neill held leadership roles of increasing responsibility in clinical research and development. From November 2016 to June 2018, Dr. O’Neill served as Biogen’s senior vice president, late-stage clinical development and oversaw development programs for Alzheimer’s disease, movement disorders, acute neurology, multiple sclerosis, pain, neuromuscular disease, gene and cell therapy, and rare diseases. He played a leadership role in seeking, receiving and maintaining global marketing approvals for Tecfidera®, Zinbryta®, Plegridy® and Spinraza®. Dr. O’Neill is a member of the American Academy of Neurology and a board-certified neurologist (ABPN), licensed to practice medicine in the state of Massachusetts. He served as director of the leukodystrophy service and chief resident in neurology during his time at MGH as full-time physician, and has maintained his clinical appointment at MGH with a sub-specialty interest in neuromuscular diseases and inherited leukodystrophies. From 1997 to 2015, he served as a clinical instructor in neurology at Harvard Medical School. Dr. O’Neill also serves on the board of directors of the Massachusetts Biotechnology Council (MassBio). Dr. O’Neill received a Bachelor of Medicine degree at University College Dublin and a Master of Medical Sciences degree from Harvard University. He is the recipient of numerous awards in science and medicine, including the Lefler Fellowship in the Department of Neurobiology at Harvard Medical School. Dr. O’Neill is the author of numerous publications on multiple sclerosis, has served as a neurology peer reviewer for medical literature, and lectures in the United States and globally on advances in neurology and neurological research.
Executive Vice-President & CEO, Ipsen North America
Richard joined Ipsen in February 2018 and brings more than 25 years of global biopharmaceutical industry experience to the company, with notable achievements for his leadership, vision, and exceptional execution in leading and growing businesses, both in the US and internationally. With a patient- focused mindset and strong track record of successfully building high- performing, customer-centric teams, Richard is responsible for driving continued growth in both the US and Canadian markets across key therapeutics areas of oncology, neurology and rare diseases. Richard serves as a member of the Executive Leadership Team and reports directly to David Meek, CEO of Ipsen.
Richard joined Ipsen from Amgen, where he held varying leadership positions across Europe and North America during his 10 years at the company. In his most recent role as Vice President and General Manager, U.S. Oncology Business Unit, Richard led numerous successful product launches in the oncology and hematology therapeutic areas. Richard also demonstrated his innate leadership ability and passion for employee growth and mentorship through the implementation of a highly successful engagement and people development strategy. Prior to leading the U.S. Oncology Business, Richard served as the Vice President of marketing for the U.S. Oncology Business, General Manager Amgen Germany and General Manager Amgen Central & Eastern Europe. Prior to Amgen, Richard held a number of global leadership positions at Pfizer, including serving as General Manager of Pfizer South Africa and Pfizer Czech Republic. Richard also held a variety of sales, marketing and market access roles with increasing seniority at Glaxo Wellcome in Canada.
Richard earned his undergraduate degree in Commerce from the University of Saskatchewan, Canada, and his MBA from the University of Toronto, Canada.
Chairman and Chief Executive Officer, Eloxx Pharmaceuticals
Mr. Robert (Bob) E. Ward is the Chairman of the Board and Chief Executive Officer of Eloxx Pharmaceuticals, Inc. (“Eloxx”). He previously served as the Chief Executive Officer and President at Radius Health, Inc., successfully completing the initial public offering that became the top performing IPO in 2014; raised over $780M from private and public sources; achieved FDA approval and launch of the new drug TYMLOS™ while also gaining Fast Track development status for the Elacestrant oncology program.
Prior to joining Radius, Mr. Ward held a series of progressive management and executive roles with established companies such as NPS Pharmaceuticals, Schering-Plough (Merck), Pharmacia (Pfizer), Bristol-Myers Squibb and Genentech. Mr. Ward has been a Director of Akari Therapeutics, Plc since October 2016 where he chairs the Governance Committee. He served as a Director of Radius from December 2013 until July 2017. Mr. Ward serves as a Director of the Massachusetts High Technology Council. Mr. Ward received a B.A. in Biology and a B.S. in Physiological Psychology, both from the University of California, Santa Barbara, an M.S. in Management from the New Jersey Institute of Technology and an M.A. in Immunology from the Johns Hopkins University School of Medicine.