RFP Closes for Spinal Muscular Atrophy Basic Research Funding

On September 5, we announced the close of our for request for proposals (RFP) for basic research.

An RFP is an invitation for scientists to submit their best ideas for SMA research. They explain what they want to study, how they plan to study it, and why they think it will be effective for SMA.

The closing of the RFP means we can move on to the next step of the process, and make sure your donations reach scientists who can make a difference for our community.

The next step is a detailed review by our Scientific Advisory Board. They’ll review each of the 29 proposals we received, looking to see which projects are the most intriguing, which have a well constructed study plan, and which match up with the most pressing unanswered questions about SMA.  

Most importantly, they are looking for the projects that show the most promise in getting us closer to our goal of a treatment and cure for SMA. Once this evaluation is complete, we determine which projects will receive a Cure SMA research grant. 

At the end of this process, in early 2015, we’ll announce up to $700,000 in new basic research funding. 

Why We Fund Basic Research

Basic research is the critical first step in the research process, and has led to many discoveries. For example, basic research is how we identified the SMN1 mutation that causes SMA. Basic research is how we identified SMN2, the SMA “back-up gene.”  

Right now, scientists are working on gene transfer techniques that may treat the SMN1 mutation. They’re working on small molecules and other methods that may help the SMN2 gene function better. Many of these projects are also receiving Cure SMA funding, and all of them started with basic research discoveries.

Without basic research, the SMA drug pipeline would not continue to grow and diversify. We need both a breadth and a depth of options in our quest for an effective SMA therapy.  Basic research is our investment in future drug development for SMA.

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