The International LAM Registry: a component of an innovative web-based clinician, researcher, and patient-driven rare disease research platform.
BACKGROUND
A relative inability to capture a sufficiently large patient population in any one geographic location has traditionally limited research into rare diseases.


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.
Nurok
Michael
M
Division of Surgical Critical Care, Cardiac and Thoracic Anesthesia, Brigham and Women's Hospital, Harvard Medical School, Boston, Massachusetts 02115, USA. mnurok@partners.org
Eslick
Ian
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Carvalho
Carlos R R
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Costabel
Ulrich
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D'Armiento
Jeanine
J
Glanville
Allan R
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Harari
Sergio
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Henske
Elizabeth P
EP
Inoue
Yoshikazu
Y
Johnson
Simon R
SR
Lacronique
Jacques
J
Lazor
Romain
R
Moss
Joel
J
Ruoss
Stephen J
SJ
Ryu
Jay H
JH
Seyama
Kuniaki
K
Watz
Henrik
H
Xu
Kai-Feng
KF
Hohmann
Elizabeth L
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Moss
Frank
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D002986:Clinical Trials as Topic; D003194:Computer-Assisted Instruction; D006801:Humans; D018192:Lymphangioleiomyomatosis / Q000175:diagnosis / Q000235:genetics / Q000628:therapy*; D035583:Rare Diseases / Q000175:diagnosis / Q000235:genetics / Q000628:therapy*; D012042:Registries