TY - JOUR
T1 - The International LAM Registry
T2 - A component of an innovative web-based clinician, researcher, and patient-driven rare disease research platform
AU - Nurok, Michael
AU - Eslick, Ian
AU - Carvalho, Carlos R.R.
AU - Costabel, Ulrich
AU - D'Armiento, Jeanine
AU - Glanville, Allan R.
AU - Harari, Sergio
AU - Henske, Elizabeth P.
AU - Inoue, Yoshikazu
AU - Johnson, Simon R.
AU - Lacronique, Jacques
AU - Lazor, Romain
AU - Moss, Joel
AU - Ruoss, Stephen J.
AU - Ryu, Jay H.
AU - Seyama, Kuniaki
AU - Watz, Henrik
AU - Xu, Kai Feng
AU - Hohmann, Elizabeth L.
AU - Moss, Frank
PY - 2010/3/1
Y1 - 2010/3/1
N2 - 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
AB - 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
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U2 - 10.1089/lrb.2009.0028
DO - 10.1089/lrb.2009.0028
M3 - Review article
C2 - 20235890
AN - SCOPUS:77949823301
SN - 1539-6851
VL - 8
SP - 81
EP - 87
JO - Lymphatic Research and Biology
JF - Lymphatic Research and Biology
IS - 1
ER -