March 1-2, 2011

Workshop Organizers


  • *A. James Barkovich, M.D.—University of California, San Francisco
  • *Stanley J. Szefler, M.D.—University of Colorado, Denver
  • Eric Olson, Ph.D.—Vertex Pharmaceuticals Incorporated
  • William Rymer, M.D., Ph.D.—Northwestern University


Ultimately, the goal of much biomedical and behavioral research is to yield new or improved diagnostics and therapeutics to prevent and treat conditions and to enhance quality-of-life.  More powerful methods and tools are needed to enable translation of basic discoveries into new or improved interventions. Diagnostics and therapeutics must also be developed or adapted to meet the needs of specific populations, including infants, children, women, and persons with disabilities, while remaining sensitive to complex developing systems. In addition, the effects of current clinical practice on these populations must be better understood to ensure that all may benefit.

As examples, discussions of this theme could include:

  • Developing more powerful research methods and tools to create novel diagnostics and therapeutics ranging from developing new imaging technologies and mobile technologies, using nanotechnologies, improving ways to manipulate stem cells and small molecules, and improving vectors for gene therapy—to improving statistical methods for small clinical trials and longitudinal research, outcome measures, and surrogate markers, and creating normative databases
  • Understanding the basic science needed to develop personalized diagnostics and therapeutics, such as enhanced understanding of pharmacokinetics, pharmacodynamics, and pharmacogenomics in infants, children, and pregnant women and a focus on bio- and tissue engineering
  • Creating the next generation of diagnostics and therapeutics targeted to specific areas of interest to the NICHD, such as novel pediatric devices, prosthetics, reengineered tissue, and an enhanced “clinical toolkit” to address intellectual and developmental disabilities and cognitive impairment, as well as an understanding of how full implementation and use of such tools could affect the health care system
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Last Reviewed: 05/23/2011