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. Guide to Spinal Muscular Atrophy Research from Families of SMA. The guide helps to answer questions such as what are the key areas of SMA research and what are the costs for conducting SMA drug development and clinical trials. Clear definitions and graphics are provided to help explain and illustrate how SMA drugs begin and the key steps involved in developing new therapies for Spinal Muscular Atrophy. The topics in this new booklet cover the following important areas of SMA research:
SMA Researchers; Drug Research; Gene Therapy; Stem Cells; Clinical Trials; and Government Research and The FDA. Click the image below to download the booklet: If you would like a hard copy mailed to you please email us at info@fsma.org or call (800) 886-1762.
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