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Spinal Muscular Atrophy (SMA) Biomarker Qualification Workshop


May 13, 2011
Silver Spring, MD

The purpose of this Spinal Muscular Atrophy (SMA) Biomarker Qualification Workshop was to explore a variety of molecular and other potential biomarkers for SMA that may be of use in future clinical trials, highlighting the current state of knowledge and the important knowledge gaps regarding each that should be addressed to strengthen their value. Discussion also included an overview of the process for biomarker qualification at FDA and regulatory candidates for qualification. The workshop proceedings are posted below.

SMA Biomarker Qualification Workshop Summary

Agenda

 

Spinal Muscular Atrophy (SMA) Biomarker Qualification Workshop Agenda
Friday, May 13, 2011

8:00am
Introduction to NeuroNEXT
Elizabeth McNeil, NIH
8:15am
Overview of SMA - "Setting the Stage"
Stephen Kolb, M.D., Ph.D., Ohio State University
8:30am
Introduction to the FDA Biomarker Qualification Program
Marc Walton, M.D., Ph.D., FDA
9:00am
Biomarkers in Drug Development
Anne Pariser, M.D., FDA

SESSION ONE: Molecular Biomarkers

9:30am
SMN Transscript Levels
F. Danilo Tiziano, M.D., Institute of Medical Genetics, Rome
10:00am
SMN Protein Levels
W. Ludo van der Pol, M.D., Ph.D., University Medical Center Utrecht
10:30am
BREAK
10:45am
Other Markers of Disease Progression (BforSMA)
Dione Kobayashi, Ph.D., Spinal Muscular Atrophy Foundation
11:15am
Panel Discussion
Speakers and FDA Representatives
11:45am
LUNCH

SESSION TWO: Electrophysiological and Other Assessments

12:15pm
CMAP/MUNE
Kathryn Swoboda, M.D., University of Utah School of Medicine
12:45pm
Electrical Impedance Myography
Seward Rutkove, M.D., Beth Israel Deaconess Medical Center
1:15pm
Strength
Thomas Crawford, M.D., Johns Hopkins Hospital
1:45pm
Imaging
Douglas Sproule, M.D., Columbia University
2:15pm
Session Two Panel Discussion
Speakers and FDA Representatives
2:15pm
Break

SESSION THREE: Next Steps

3:00pm
General Discussion of Selected Candidates
  • Potential utility
  • Strength of evidence
  • Gaps to be filled
  • Aspects of drug development
4:30pm
Closing Comments/Adjourn
Elizabeth McNeil, M.D., M.Sc., NIH

Last updated March 20, 2013