Latest News
Scholar Rock Reports Preliminary Pharmacokinetic and Pharmacodynamic Data from TOPAZ Phase 2 Trial of SRK-015 for the Treatment of Patients with Spinal Muscular Atrophy
Scholar Rock today announced preliminary pharmacokinetic (PK) and pharmacodynamic (PD) results from the TOPAZ Phase 2 proof-of-concept trial of SRK-015 for the treatment of patients with spinal muscular atrophy (SMA). […]
Read More ›Team Cure SMA Raises Over $45,000 in the New York City Marathon
Fifteen Team Cure SMA runners successfully raised over $45,000 for Cure SMA while running in the 2019 TCS New York City Marathon on November 3, 2019. The all-star team included: […]
Read More ›Genentech’s Risdiplam Meets Primary Endpoint in Pivotal SUNFISH Trial in People With Type 2 or 3 Spinal Muscular Atrophy
Genentech, a member of the Roche Group, today announced positive data from the pivotal Part 2 of the SUNFISH study evaluating risdiplam in people aged 2-25 years with Type 2 […]
Read More ›A Tiny Warrior: Winnie Stanley’s Story
This guest post is written by April Leiffer Henry If you ask 4-year-old Winnie Stanley what she wants to be when she grows up, she’ll say a superhero. But to […]
Read More ›Community Spotlight: Angela Wrigglesworth
October has been National Disability Employment Awareness Month, and throughout the month, you have heard stories about adults in the workforce who live with SMA. We are pleased to close […]
Read More ›AveXis Issues Community Statement on AVXS-101 Clinical Hold
Dear SMA community, Today, we announced an AVXS-101 intrathecal study update. In the update, we shared that the United States Food & Drug Administration (FDA) placed a partial hold on […]
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