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Funding basic research ongoing in academic labs at Universities across the world serves as our long-term investment in building the SMA therapeutics pipeline.
Basic research projects identify the most effective new strategies for SMA drug discovery. Essentially, basic research fuels the SMA therapeutics pipeline by creating innovation and revealing new ways to make SMA drugs, including alternative possibilities such as stem cell therapies and anti-sense nucleotide therapies. Without basic research our SMA drug pipeline would not continue to grow and diversify, which is critical to ensuring we take every possible shot on goal in our quest for an effective SMA therapy. Therefore, at FSMA we view basic research as our investment in the future.
To make the best possible SMA therapies questions about SMA pathology need to be answered, including:- In what tissues is SMN lacking to cause SMA?
- What is the critical function of SMN in SMA?
- How late in disease progression can SMN be replaced and provide benefit?
- What molecular targets besides SMN2 can serve as the basis for a SMA drug?
- Can increasing muscle strength alone without improving MN function provide therapeutic benefit in SMA animal models?
Each year FSMA issues a call for grant applications that address specific unanswered research questions in SMA biology. The topics included in our call for grant applications is developed in concert with our Scientific Advisory Board during a dedicated meeting at the Annual International SMA Research Group Meeting. Several months later the applications are reviewed by our SAB to select those to be funded by FSMA. Over the last 25 years FSMA basic research funding has contributed to many critical SMA breakthroughs, including the following:- Identification of the SMN protein and its roles in the cell
- Discovery of the back-up SMA gene, SMN2
- Development of multiple animal models for SMA
- Finding HDAC inhibitors enhance SMN2 gene expression
- Identification of the nucleic acid sequence used to correct SMN2 splicing by ISIS Pharamceuticals
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