SMA Biomarker Study

In the Spinal Muscular Atrophy (SMA) Biomarker Study, we hope to learn how babies with SMA develop compared to their age matched peers. Researchers have discovered that there is a critical time period when treatment is effective in SMA mice. In mice, this time period is immediately after birth. The same therapy given to the mice at a later time period is much less effective. At this time, no one knows if such a critical time period exists in humans. In this study, we will focus on infants with SMA to determine whether this critical time point exists in babies. That’s why we need both SMA and healthy infants younger than 6 months of age to help us determine:

  • When treatments in SMA should be given 
  • What happens to SMN protein levels as children grow
  • What tests will be required for SMA clinical trials of the future

You and your child will come to a study site for regular study visits based on your child’s age. At these visits a variety of procedures will be performed, including:

  • Motor function tests
  • Non-invasive tests to measure the function of nerves and muscles
  • A blood draw that is performed on most, but not all, of the study visits

If you are interested in participating, find more information in the study brochure​ or call 1.855.762.2466.