Fitzpatrick Institute for Photonics Fitzpatrick Institute for Photonics
Pratt School of Engineering
Duke University

 HOME > pratt > FIP    Search Help Login pdf version printable version 

Publications [#192794] of James M Provenzale

Papers Published

  1. PI Dickson, AR Pariser, SC Groft, RW Ishihara, DE McNeil, D Tagle, DJ Griebel, SG Kaler, JW Mink, EG Shapiro, KJ Bjoraker, L Krivitzky, JM Provenzale, A Gropman, P Orchard, G Raymond, BH Cohen, RD Steiner, SF Goldkind, RM Nelson, E Kakkis, MC Patterson, Research challenges in central nervous system manifestations of inborn errors of metabolism., Molecular genetics and metabolism, vol. 102 no. 3 (March, 2011), pp. 326-38, ISSN 1096-7206 [doi]
    (last updated on 2011/07/12)

    Abstract:
    The Research Challenges in CNS Manifestations of Inborn Errors of Metabolism workshop was designed to address challenges in translating potential therapies for these rare disorders, and to highlight novel therapeutic strategies and innovative approaches to CNS delivery, assessment of effects and directions for the future in the treatment of these diseases. Therapies for the brain in inborn errors represent some of the greatest challenges to translational research due to the special properties of the brain, and of inborn errors themselves. This review covers the proceedings of this workshop as submitted by participants. Scientific, ethical and regulatory issues are discussed, along with ways to measure outcomes and the conduct of clinical trials. Participants included regulatory and funding agencies, clinicians, scientists, industry and advocacy groups.

    Keywords:
    Animals • Biomedical Research* • Central Nervous System* • Clinical Trials as Topic • Humans • Metabolism, Inborn Errors • Rare Diseases • diagnosis* • ethics • pathology • physiopathology • therapy • therapy* • trends


Duke University * Pratt * Reload * Login