August is SMA Awareness Month. Through research supported by Cure SMA, scientists have discovered SMN2, the SMA “backup gene.” Most of the SMN protein produced by SMN2 lacks a key building block that is normally produced by SMN1, but people with more copies of SMN2 sometimes have a less severe form of SMA than those with fewer copies. Researchers are working to develop drugs that may prompt SMN2 to make more protein.
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