The International LAM Registry: A component of an innovative web-based clinician, researcher, and patient-driven rare disease research platform

Michael Nurok, Ian Eslick, Carlos R.R. Carvalho, Ulrich Costabel, Jeanine D'Armiento, Allan R. Glanville, Sergio Harari, Elizabeth P. Henske, Yoshikazu Inoue, Simon R. Johnson, Jacques Lacronique, Romain Lazor, Joel Moss, Stephen J. Ruoss, Jay H. Ryu, Kuniaki Seyama, Henrik Watz, Kai Feng Xu, Elizabeth L. Hohmann, Frank Moss

Research output: Contribution to journalReview article

14 Scopus citations

Abstract

Background: A relative inability to capture a sufficiently large patient population in any one geographic location has traditionally limited research into rare diseases. Methods and Results: Clinicians interested in the rare disease lymphangioleiomyomatosis (LAM) have worked with the LAM Treatment Alliance, the MIT Media Lab, and Clozure Associates to cooperate in the design of a state-of-the-art data coordination platform that can be used for clinical trials and other research focused on the global LAM patient population. This platform is a component of a set of web-based resources, including a patient self-report data portal, aimed at accelerating research in rare diseases in a rigorous fashion. Conclusions: Collaboration between clinicians, researchers, advocacy groups, and patients can create essential community resource infrastructure to accelerate rare disease research. The International LAM Registry is an example of such an effort.82

Original languageEnglish (US)
Pages (from-to)81-87
Number of pages7
JournalLymphatic Research and Biology
Volume8
Issue number1
DOIs
StatePublished - Mar 1 2010

ASJC Scopus subject areas

  • Cardiology and Cardiovascular Medicine

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    Nurok, M., Eslick, I., Carvalho, C. R. R., Costabel, U., D'Armiento, J., Glanville, A. R., Harari, S., Henske, E. P., Inoue, Y., Johnson, S. R., Lacronique, J., Lazor, R., Moss, J., Ruoss, S. J., Ryu, J. H., Seyama, K., Watz, H., Xu, K. F., Hohmann, E. L., & Moss, F. (2010). The International LAM Registry: A component of an innovative web-based clinician, researcher, and patient-driven rare disease research platform. Lymphatic Research and Biology, 8(1), 81-87. https://doi.org/10.1089/lrb.2009.0028