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 novel kinds of SMA drug candidates. Establishing clinical trial infrastructure provides the means to test the drug candidates in humans. Therefore, continued investment in all three research areas is essential to finding an effective SMA treatment and ultimate 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; Antisense Oligonucleotides, Clinical Trials; Government Research: and The FDA.
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