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

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.

journal_name

Lymphat Res Biol

authors

Nurok M,Eslick I,Carvalho CR,Costabel U,D'Armiento J,Glanville AR,Harari S,Henske EP,Inoue Y,Johnson SR,Lacronique J,Lazor R,Moss J,Ruoss SJ,Ryu JH,Seyama K,Watz H,Xu KF,Hohmann EL,Moss F

doi

10.1089/lrb.2009.0028

subject

Has Abstract

pub_date

2010-03-01 00:00:00

pages

81-7

issue

1

eissn

1539-6851

issn

1557-8585

journal_volume

8

pub_type

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