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The ultimate goal at Families of SMA is to accelerate the discovery of an effective treatment and cure for SMA. To reach this end, our research program has three distinct parts: - basic research to unravel the biology of SMA,
- pre-clinical drug discovery to develop SMA drugs, and
- human clinical trial initiatives to test these new drugs.
We are also making substantial investments in promising alternative therapies, such as stem cell therapy to diversify and so improve our chances for success.
All three of these research areas are equally critical and are interdependent. Basic research in SMA biology tells us what causes SMA. Understanding what causes SMA reveals new and more effective ways of making SMA drugs. Pre-clinical drug discovery programs then convert basic research ideas into new, better, and more directed SMA drug candidates. Establishing clinical trial infrastructure provides the means to test these new drug candidates in humans.
Therefore, continued investment in all these research areas is essential to finding an effective SMA treatment and ultimately a cure.
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