Latest News
Scholar Rock Announces Positive Interim Results from Phase 1 Trial of SRK-015 in Healthy Volunteers and Updates on Future Development Plans
Scholar Rock today announced positive interim results from the ongoing Phase 1 single- and multiple-ascending dose clinical trial of SRK-015 in healthy adult volunteers. SRK-015, a highly specific inhibitor of […]
Read More ›Community Spotlight: Kristen Smith
The SMA community has been lobbying state and federal government for years. Kristen Smith shares how she became involved with advocacy. Kristen Smith describes herself as an artist and advocate. […]
Read More ›Arkansas and Florida Adopt SMA Newborn Screening
We are thrilled to share that both Arkansas and Florida recently adopted SMA to their state newborn screening panels! They now join the growing list of states that have adopted […]
Read More ›Cure SMA Awards $150,000 Grant to Veronica Pessino, PhD, Salk Institute for Biological Sciences
Audrey Lewis founded Families of SMA, now Cure SMA, 34 years ago. Audrey recognized early on the importance of attracting new and talented researchers to SMA, with the hope that […]
Read More ›Community Spotlight: Amber Bosselman
Amber Bosselman is your typical 22-year-old college senior. She is currently studying psychology in Idaho and planning to get her master’s degree in industrial psychology. Amber’s life is similar to […]
Read More ›Show Your Rare. Show You Care.
1 in 20 people will live with a rare disease at some point in their life. Rare Disease Day improves knowledge amongst the general public of rare diseases while encouraging […]
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