may, 2017

25may4:30 pm7:00 pmAdvancing the Development of Rare Disease Therapies


Event Details

Advancing the Development of Rare Disease Therapies

There are approximately 7,000 rare diseases affecting 1:10 Americans currently; however 95 percent of rare diseases do not have an FDA approved treatment. Industry interest in rare diseases has grown in recent years due to market interest and regulatory incentives from the FDA. Boston Children’s Hospital and its Manton Center for Orphan Disease Research are at the forefront of conducting research to identify, understand, diagnose and treat these diseases. We invite you to learn more about Boston Children’s research advances in rare diseases, hear from our researchers in hematology, oncology, pulmonary, cardiology, neurology and more, and to discuss advancing new therapies through powerful industry/academic collaborations.


  • 4:30 registration & networking – cocktails and hors d’oeuvres
  • 5:00 welcome & panel discussion
  • 5:40 research talks
  • 6:00 networking session with posters

Panel Discussion – Bridging the gap in rare disease therapeutics development between academia and industry


  • David Williams, MD, Chief Scientific Officer, Boston Children’s Hospital.
  • Alan Beggs, PhD, Director, The Manton Center for Orphan Disease Research,Boston Children’s Hospital.
  • Philip Reilly, MD, JD, Venture Partner, Third Rock Ventures.
  • Alvin Shih, MD, MBA, ‎CEO, Enzyvant.

This event is the first of three scientific networking events organized by Boston Children’s Hospital and hosted by MassBio. These events will highlight the latest and greatest discoveries from Boston Children’s Hospital, cultivate community engagement and facilitate collaborations between scientists from Boston Children’s Hospital and the life science industry.

This event is brought to you by Boston Children\’s Hospital\’s Technology and Innovation Development Office and the Manton Center for Orphan Disease Research.


(Thursday) 4:30 pm - 7:00 pm


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300 Technology Square, Cambridge, MA 02139