Join MassBio in Supporting Rare Disease Research

Feb 15, 2017

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.

Please consider joining us for the following MassBio events to support the cause! 

Role of Patient Advocacy Organizations in Advocating for Drug Approvals
Tuesday, February 28th
9:00 am – 10:30 am 
Massachusetts State House, Great Hall

Our Rare Disease Day panel will take into consideration the complex nature of rare disease therapy research and development and discuss: 

  • The evolving role of patient advocacy organizations in overall drug development
  • How patients fit in research and development
  • Current regulatory agencies' initiatives on utilizing patients' input in drug review and approval process
  • The use and limitations of data collected from online communities as part of regulatory approval process


  • John K. Jenkins, MD, Principal, Drug and Biological Products, Greenleaf Health, Former Director, FDA Office of New Drugs
  • Sarah Lieber, Head of Rare Disease Therapeutic Area, Global Regulatory Affairs, Sanofi Genzyme
  • Gilmore O'Neill, MD, Senior Vice President, Late Stage Clinical Development, Biogen
  • Tracy Seckler, Co-Founder and Chief Visionary Officer, Charley's Fund
  • Sara Singleton, Senior Director of Research, Drug Discovery and Development Policy, Cystic Fibrosis Foundation
  • Kate Rawson, The RPM Report: Regulation, Policy and Market Access and “The Pink Sheet”, Prevision Policy LLC (Moderator)


Rare Disease Day 2017: Patient Involvement in Research
Tuesday, February 28th
11:00 am – 12:00 pm
Massachusetts State House, Great Hall

The complex nature of rare diseases, coupled with limited access to treatment and services, means that family members are often the primary source of solidarity, support and care for their loved ones. The Rare Disease Day 2017 theme Patient Involvement in Research focuses on the crucial role that patients play in research. Patients are often experts in their disease. It is essential that research also involves patients at all steps of the research process. Rare Disease Day 2017 is therefore an opportunity to call upon researchers, universities, students, companies, policy makers and clinicians to do more research and to make them aware of the importance of research for the rare disease community.

Speakers include:

  • Representative Jennifer Benson, Massachusetts House of Representatives
  • Katie Brandt, Caregiver and Community Resource Specialist, Frontotemporal Disorders Unit,
    Massachusetts General Hospital (Emcee)
  • Mark Borreliz, Lawyer with Hemophilia A
  • Tim Boyd, Associate Director of State Policy, National Organization for Rare Disorders (NORD)
  • Janis Creedon, Mother of a 5 year old son with an ultra-rare chromosome abnormality, undiagnosed rare disease
  • Elaine Kiriakopoulos, MD, MSc, Chief Operating Officer, Epilepsy Foundation New England
  • Patrick Lacey, President & Founder, Beat NB, and father of a neuroblastoma survivor
  • Justin Peavey, Advocate for awareness of Frontotemporal Degeneration (FTD)

Please join us to recognize Rare Disease Day and the research being done in Massachusetts to treat and cure rare disease. Together, we can raise awareness of rare disease.


We also want to thank our Rare Disease Day sponsors: 

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