2012 Conference Resources

Abstract and Poster Session

Conference attendees are encouraged to submit an abstract which are then reviewed by the planning committee. The abstracts selected by the planning committee were invited to be displayed as a poster during the conference.

Investigators working on rare diseases provided feedback and advice during the poster session.

PDF Viewer is required.

2012 CCRRD Presentations Videos and Slides

Welcome and Introduction
Peter A. Merkel, MD, MPH; Stephen C. Groft, PharmD
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Clinical Trial Design Issues and Options for the Study of Rare Diseases
Jeffrey Krischer, Ph.D
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Ethical Issues in Research on Rare Diseases
David Wendler
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Development of a CFTR Potentiator The Perspective of a Clinical Investigator
Bonnie W. Ramsey, MD
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Vertex Program to Discover and Develop Therapies for Cystic Fibrosis
Eric Olson, PhD
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Do Anti-Phospholipase A2 Receptor Antibodies Predict Recurrence of Membranous Nephropathy After Transplantation?
Rivka Ayalon MD
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Idiopathic Bronchiectasis and Connective Tissue Fibrillinopathies: Dural Ectasia as a Marker of a Distinct Bronchiectasis Subgroup
M. Leigh Anne Daniels, MD, MPH
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Exome sequencing and functional validation in zebrafish identify novel genes for dystroglycanopathies
M. Chiara Manzini PhD
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Cholesterol pool size in Smith-Lemli-Opitz syndrome children receiving cholesterol supplementation alone or combined with simvastatin
Semone B. Myrie
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2010 Conference Resources

Abstract and Poster Session

Conference attendees are encouraged to submit an abstract which are then reviewed by the planning committee. The abstracts selected by the planning committee were invited to be displayed as a poster during the conference.

Investigators working on rare diseases provided feedback and advice during the poster session.

PDF Viewer is required.

2010 CCRRD Presentations Videos and Slides

Welcome and Introduction
Peter A. Merkel, MD, MPH; Stephen C. Groft, PharmD
[ Watch Video ]

Creating, Maintaining, and Growing Clinical Research Networks in Rare Diseases
Peter A. Merkel, MD, MPH
[ Watch Video ] [ Download Slides ]

Clinical Trial Design Issues and Options for the Study of Rare Diseases
Jeffrey Krischer, PhD
[ Watch Video ] [ Download Slides ]

The CTSA Program and Rare Disease Research
Robert D. Steiner, MD
[ Watch Video ] [ Download Slides ]

Study Designs for Rare Diseases
Keith Hull, MD, PhD
[ Watch Video ] [ Download Slides ]

Industry Perspective of Orphan Diseases Drug Development
Edward M. Kaye, MD
[ Watch Video ] [ Download Slides ]

Conflicts of Interest and Rare Diseases
Susan H. Ehringhaus
[ Watch Video ] [ Download Slides ]

Parent, Advocate, Researcher (The View From My Parachute)
Jannine D. Cody, PhD
[ Watch Video ] [ Download Slides ]

2007 Conference Resources