2020 Rare Disease Day
February 28, 2020 9:30 AM - 2:00 PM
Omni Parker House, 60 School St Boston MA 02108
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2/28/2020 9:30:00 AM
2/28/2020 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
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
Former Founder, President & CEO | X4 Pharmaceuticals
Paula Ragan, Ph.D. is a seasoned leader focused on growing companies and driving
innovation with over 25 years of experience in the biotechnology and life sciences industry.
She is currently the Managing Director of PMR Advisory LLC, a consulting firm that partners
with privately held biotechnology companies to advance their strategic goals. Most
recently, Paula founded and served as Chief Executive Officer of X4 Pharmaceuticals, Inc.,
building the company from inception to a successful initial public offering and advancing
its lead asset to FDA approval and U.S. launch as XOLREMDI® (mavorixafor), a first-in-class
Breakthrough Therapy for a rare primary immunodeficiency. Before founding X4, Paula
served as consulting Chief Business Officer at Lysosomal Therapeutics. Earlier in her
career, she spent significant time at Genzyme Corporation, where she held leadership
roles in corporate development, manufacturing, and supply chain.
Currently, Paula is member of the Board of Advisors for Life Science Cares and a member
of the Landmark School Fundraiser Committee. Additionally, Paula serves as a mentor for
the Termeer Institute, a non-profit organization cultivating the next generation of biotech
leaders. Previously, Paula also served as a Director on the Board of Camp4 Therapeutics.
Paula holds a Ph.D. in Medical Engineering and Medical Physics from the Harvard-MIT
Division of Health Sciences and Technology (HST), an M.S. in Biomedical Engineering from
Boston University, and a B.S. in Mechanical Engineering from Tufts University.
