The motivation and rationale for project 1 derives from the increasing recognition that ALS and related disorders represent syndromes with shared pathophysiological mechanisms, but a multitude of different (often genetic) causes. This realization is prompting an increasing interest in the ideas that one treatment may not be appropriate for all patients with each clinical syndrome, but rather that different therapeutic approaches might be needed depending on the underlying etiology of disease. It is against this background that defining the temporal course of disease and delineating the variability in rates of disease progression in genetically homogeneous patient populations has emerged as an essential stes in preparing for the future of clinical trials in ALS and related disorders. The principal goal of Project-1 of this RDCRC is to define the natural history (i.e. the temporal rate of disease progression) of disease in patients with ALS and related disorders who harbor different underlying genetic mutations;A secondary goal is to characterize the spatial and temporal patterns of disease progression within these individual disease categories, with a focus on ALS.

Public Health Relevance

Successful completion of this project will shed significant light on the phenotypic correlates of genotype in ALS and related disorders, thereby empowering future therapeutic and disease prevention trials for patients with this group of neurodegenerative disorders in whom the underlying genetic etiology has been identified. These studies will also elucidate the pattern(s) with which disease progresses through the nervous system.

Agency
National Institute of Health (NIH)
Institute
National Institute of Neurological Disorders and Stroke (NINDS)
Type
Specialized Center--Cooperative Agreements (U54)
Project #
1U54NS092091-01
Application #
8929487
Study Section
Special Emphasis Panel (ZTR1-CI-8 (01))
Program Officer
Gubitz, Amelie
Project Start
2014-09-30
Project End
Budget Start
2014-09-30
Budget End
2015-06-30
Support Year
1
Fiscal Year
2014
Total Cost
$325,372
Indirect Cost
$101,260
Name
University of Miami School of Medicine
Department
Type
DUNS #
052780918
City
Coral Gables
State
FL
Country
United States
Zip Code
33146
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