Research
Cure SMA Launches “Spotlight on SMA” Partnership with Neurology Reviews
Cure SMA is pleased to announce the release of “Spotlight on SMA: The Urgent Need for Early Diagnosis in Spinal Muscular Atrophy,” a supplement developed […]
Read More ›Genentech’s Risdiplam Showed Significant Improvement in Motor Function in People Aged 2-25 Years with Type 2 or Type 3 SMA
Genentech, a member of the Roche Group, today presented 1-year data from the pivotal Part 2 of the SUNFISH study, evaluating risdiplam in people aged […]
Read More ›Genentech’s Risdiplam Meets Primary Endpoint in Pivotal FIREFISH Trial in Infants with Type 1 SMA
Genentech, a member of the Roche Group, today announced positive topline results from the pivotal Part 2 of the FIREFISH study, evaluating risdiplam in infants […]
Read More ›Cure SMA and Biogen to Co-Host Webinar on DEVOTE Clinical Trial – Register Today!
On Tuesday, February 4th, at 11am CST (9am PST/10am MST/12pm EST) Biogen and Cure SMA will co-host a webinar for the SMA community. During the […]
Read More ›A Look Back at 2019: Cure SMA Research Year-in-Review
During our last fiscal year—July 1, 2018, to June 30, 2019—Cure SMA funded more than $7 million in new research and ongoing research. The resources […]
Read More ›Biogen Issues Q4 2019 Community Statement on Spinraza
Dear Members of the SMA Community, As part of Biogen’s ongoing commitment to individuals living with, and caregivers affected by, spinal muscular atrophy (SMA), we […]
Read More ›Fall 2019 Issue of Compass Now Available
The Fall 2019 issue of Compass is now available online. In this issue we provide a one-year update on the roll-out of the Cure SMA […]
Read More ›Check Out the Cure SMA Booklet on “Scientific Considerations for Drug Combinations”
There are now two treatments approved by the U.S. Food and Drug Administration (FDA) that target the underlying genetics of spinal muscular atrophy (SMA) and […]
Read More ›SMA Concordance Among Siblings Published in the Journal of Neuromuscular Diseases
Earlier this month, an article was published in the Journal of Neuromuscular Diseases titled, “Spinal Muscular Atrophy (SMA) Subtype Concordance in Siblings: Findings from the […]
Read More ›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 […]
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