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Families of SMA Releases a Request for Proposals on Research Projects for Spinal Muscular Atrophy.
 
July 9, 2014.

Program Overview: The deadline for submission is September 5, 2014. Applications will be accepted for one or two years of funding with a maximum funding level of $75,000 per year, with an overall award total of $150,000 (including 8% overhead) over the duration of the project. Applications will be accepted from laboratory PIs or post-doctoral fellows.

Scientific Priorities: Families of SMA would like to encourage applications on novel research that will enhance our understanding of SMA disease pathology at the molecular, cellular, and biochemical level; generate key reagents and tools to facilitate drug development and clinical trials; and identify new therapeutic strategies for SMA.  

Studies funded by our Basic Research RFP will fall into one the following key areas, which are listed in random order:

1. Studies focused on the molecular and biochemical mechanisms regulating SMN expression or mediating SMN function. Results should lead to a better understanding of the requirements for SMN protein biologically, informing therapy development. Such studies may also identify genetic modifiers, upstream regulators of SMN expression / splicing / function, and downstream effectors of SMN functional activity, resulting in novel drug targets. 
   
2.  Studies resulting in greater understanding of the pathophysiology of SMA, using well-validated animal or cellular models of the SMA. This includes focus on the tissue requirements for SMN protein, clarifying the cellular autonomy of the disease in motor neurons and other cells, peripheral versus central manifestations of the disease, and other areas. 

3. Early proof-of concept assessment of novel therapeutic approaches for SMA in well-validated animal or cellular models of the disease.

4. Work focused on generating research and clinical trial tools, such as new animal models for SMA, phenotypic cellular assays for SMA, biomarkers or outcome measures for SMA clinical trials, newborn screening protocols, and others.

Application Process: Please submit an electronic copy as well as 4 paper copies to Families of SMA.  The electronic copy should be sent to jill@fsma.org - include the PI’s surname and Basic Research in the subject line.  Paper copies should be sent to:

Families of SMA
925 Busse Road
Elk Grove Village, IL 60007

Review Criteria:  All applications are reviewed by our Scientific Advisory Board (SAB), on both scientific quality and relevance to the Families of SMA research mission of accelerating the discovery of safe and effective treatments for SMA.

Timeline:
RFP Release Date:                                   July 9, 2014
Proposal Due Date:                                  September 5, 2014
Award Notification:                                   January 31, 2015 

For more information or if you have questions, please contact Jill Jarecki PhD, FSMA Research Director at jill@fsma.org

 

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  • 2014 FSMA Grant Application

     

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