Research
Cure SMA Launches Third Annual Community Survey to Address Important Issues in SMA Treatment
For the past several years, we’ve been working to collect data and information on our community’s experiences, goals, hopes, and challenges. We know that the […]
Read More ›Recording of Genentech/Roche and Cure SMA Webinar Now Available Online
A recording of this week’s webinar with Genentech/Roche is now available online. During the webinar, Genentech/Roche shared their history working with the SMA community, and […]
Read More ›Cure SMA Advocates for SMA Community at ICER Hearing
On Thursday, March 7, Cure SMA testified on behalf of the SMA community at a meeting of the Institute for Clinical and Economic Review (ICER). […]
Read More ›Biogen Provides 2019 Community Update on Spinraza
Dear members of the SMA community, As we celebrate Rare Disease Day 2019, we are honored to continue supporting the spinal muscular atrophy (SMA) community. […]
Read More ›Genentech/Roche and Cure SMA to Co-Host Webinar
On Monday, March 11th, at 12pm CST (10am PST/11am MST/1pm EST) Genentech/Roche and Cure SMA will co-host a webinar for the SMA community. During the […]
Read More ›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, […]
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 […]
Read More ›Cure SMA Awards $150,000 Grant to Laxman Gangwani, PhD, Texas Tech University
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 […]
Read More ›Cytokinetics Announces Receipt of FDA Feedback Regarding Reldesemtiv in Patients with SMA
Cytokinetics, Incorporated (CYTK) today announced that it has received feedback from the U.S. Food and Drug Administration (FDA) that the Six Minute Walk Test (6MWT) […]
Read More ›Genentech, Roche and PTC Therapeutics Provide Update on Regulatory Filing for Risdiplam
On January 7th, PTC Therapeutics, Inc. provided a corporate update, which will be detailed as part of the company’s presentation at the 37th Annual J.P. […]
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