Scholar Rock Announces Positive Interim Results from Phase 1 Trial of SRK-015 in Healthy Volunteers and Updates on Future Development Plans

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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 myostatin activation, was observed to be well tolerated with no apparent safety signals. By demonstrating robust and sustained target engagement in humans, pharmacologic effects on […]

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Community Spotlight: Kristen Smith

Leave a Comment / Advocacy, Community Awareness, Community Spotlight, Front Page News /

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. A powerful combination during a time where people across the country are standing up for their community and speaking out for their rights. Kristen is

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Arkansas and Florida Adopt SMA Newborn Screening

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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 SMA to their newborn screening panels. Arkansas Adopts SMA Newborn Screening Congratulations to Arkansas, who just passed legislation to add SMA to their newborn screening

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Cure SMA Awards $150,000 Grant to Veronica Pessino, PhD, Salk Institute for Biological Sciences

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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 they would commit their careers to developing a treatment and cure for SMA. Cure SMA honors Audrey’s legacy with the Audrey Lewis Young Investigator Award,

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Community Spotlight: Amber Bosselman

Leave a Comment / Community Awareness, Community Spotlight, Conference, Front Page News /

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 other students, except she was born with a rare disease called spinal muscular atrophy (SMA). SMA is a disease that robs people of strength by

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Cure SMA Awards $150,000 Grant to Laxman Gangwani, PhD, Texas Tech University

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Cure SMA has awarded a $150,000 research grant to Laxman Gangwani, PhD, at the Texas Tech University Health Sciences Center, for his project entitled, “Function of Senataxin as a Protective Modifier of Spinal Muscular Atrophy”. Dr. Gangwani and his team will study how the expression of the Senataxin protein may serve as a protective genetic

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Register for Cure SMA’s Advocacy 101 Webinar: Learn How to Make a Difference

Leave a Comment / Advocacy, Community Awareness, Front Page News /

On Wednesday, February 20th at 12pm CST (10am PST/11am MST/1pm EST) Cure SMA’s Policy and Advocacy team will hold a webinar to update the SMA community on the current advocacy priorities. Advocacy can be confusing and intimidating, but it is critical to making sure that policymakers hear the voice of the SMA Community. Topics covered

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