Center for Orphan Disease Research and Therapy: Faculty Retreat Feedback and Survey
Dear Registered Retreat/Center Member:
We would appreciate feedback from the First Annual Faculty Retreat, "Charting Paths Forward", held at the University of Pennsylvania Museum of Archeology and Anthropology November 21, 2013.
The goal of this survey is to provide us with the necessary feedback on how we can better work together by providing assistance in your rare disease research program.
Please ONLY rate speakers you heard during the day. The survey should take no longer than 5 minutes to complete.
Should you ever have any questions, please do not hesitated to contact my office. All responses will be kept confidential.
Associate Director, Center for Orphan Disease Research and Therapy
I was able to attend some or all of the days activities. *
What Activities did you attend? *
This section asks questions that include:
- Your over all thoughts about the day
- Feedback only on Speakers you heard
- Proposed aspects of drug/small molecule screening
- Core services the center could augment that would be useful to the rare disease community.
Overall how would your rate the retreat? *
NOTE: Please only provide feedback on speakers you heard. Thank you!
John F. Crowley, JD, MBA
Chairman and CEO, Amicus Therapeutics, Inc.
“Industry and Patient Groups Collaborating for Success in Rare Diseases”
Stephen C. Groft, Pharm.D.
Director, Office of Rare Disease Research, NIH
“Challenges and Opportunities in Rare Disease Research”
John C. McKew, Ph.D.
Acting Scientific Director, Division of Preclinical Innovation, National Center for Advancing Translational Science, NIH
“NCATS: Catalyzing Innovation”
President and CEO, National Disease Research Interchange
John Lonsdale, Ph.D.
Vice President, Partnership Development, NDRI
“NDRI Rare Disease Biospecimen Program”
Larry Gold, Ph.D.
Chairman, Founder, and CEO, SomaLogic, Inc.
“Drugs, Biomarkers, & Knowledge for the Rare Disease Community”
Cristina Csimma, PharmD, MHP
CEO, Cydan Development, Inc.
“Translating Compounds into Therapeutics: Drug Development and
Garret A. FitzGerald, M.D.
“ITMAT Resources of Relevance to CODRT”
David C. Schultz, Ph.D.
Facility Director, Molecular Screening Facility, Wistar Institute
“The Wistar Molecular Screening Facility: A Shared Resource for Early
Stage Discovery with Translational Implications for Rare Diseases”
James M. Wilson, M.D., Ph.D.
Professor of Pathology and Laboratory Medicine
“Gene Therapy Opportunities and Resources for Rare Diseases”
Jean Bennett, M.D., Ph.D.
F.M. Kirby Professor of Ophthalmology
“Seeing the Light: Gene Therapy Resources at CHOP”
Rick Wagner, Ph.D.
Founder and CEO, X-CHEM Pharmaceuticals
"X-Chem DNA-Encoded Library Technology - New Paradigm in Drug Discovery"
Carl Morris, Ph.D.
Director, Protein Therapeutics and Muscle Biology Pfizer Rare Disease Research Unit
“Rare Disease Program at Pfizer”
Mark Haskins, BA, VMD, MS, Ph.D.
Professor, University of Pennsylvania School of Veterinary Medicine