2020 Rare Disease Day

February 28, 2020 9:30 AM - 2:00 PM

Omni Parker House, 60 School St Boston MA 02108

Add to Calendar 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
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.

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