The International LAM Registry: A Component of an Innovative Web-Based Clinician, Researcher, and Patient-Driven Rare Disease Research Platform
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Nurock-2010-International LAM Registry.pdf
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111.16 KB
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Author(s)
Nurok, Michael
Eslick, Ian Scott
Carvalho, Carlos R. R.
Costabel, Ulrich
D'Armiento, Jeanine
Glanville, Allan R.
Harari, Sergio
Henske, Elizabeth P.
Inoue, Yoshikazu
Johnson, Simon R.
Date Issued
March 2010
Journal
Lymphatic Research and Biology
Publisher
Mary Ann Liebert, Inc.
Citation
Nurok, Michael et al. "The International LAM Registry: A Component of an Innovative Web-Based Clinician, Researcher, and Patient-Driven Rare Disease Research Platform." Lymphatic Research and Biology. March 2010, 8(1): 81-87. © 2010 Mary Ann Liebert, Inc.
Version
Final published version
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.
MIT Department
Massachusetts Institute of Technology. Media Laboratory
Program in Media Arts and Sciences (Massachusetts Institute of Technology)
Terms of Use
Article is made available in accordance with the publisher's policy and may be subject to US copyright law. Please refer to the publisher's site for terms of use.
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DOI of Published Version
https://doi.org/10.1089/lrb.2009.0028
