Search Results: spinraza
SMA Treatment Delays FAQ
Make today a breakthrough. Last Updated: 10/01/25 We know our community has many pressing questions related to the FDA approval delays of Scholar Rock’s […]
Read More ›Cure SMA Update on SMA Treatment Delays
Dear SMA Community, Yesterday was a disappointing and frustrating day for our community. We learned of two FDA approval delays for Scholar Rock’s apitegromab and […]
Read More ›Biogen Receives News on Supplemental New Drug Application (sNDA) for High Dose Nusinersen from the U.S. FDA
Today, Biogen announced that the U.S. Food and Drug Administration (FDA) has delayed the approval with a Complete Response Letter (CRL) for the supplemental New Drug […]
Read More ›Biogen Announces Advancement of Investigational SMA Drug Salanersen to Registrational Trials Based on Positive Phase 1 Results
“To see a child dosed with gene therapy at age one and still unable to sit at age five then gain that ability within three […]
Read More ›Biogen Announces that FDA and EMA Accept Applications for Higher Dose Regimen of Nusinersen in SMA
“We are committed to supporting individuals with SMA and their families by advancing research that aims to answer critical questions for the community.” Today, Biogen […]
Read More ›New Higher Dose Nusinersen Efficacy and Safety Data Presented at World Muscle Society Congress, Highlight Potential to Maximize Benefit of Nusinersen in SMA
Findings from Part B and Part C of the DEVOTE study support the clinical benefits of a higher dose regimen of nusinersen (50/28 mg) in […]
Read More ›Biogen Announces Positive Topline Results from Study of Higher Dose Regimen of Nusinersen, Showing Significant Benefit in Treatment of SMA
The positive study demonstrates the potential for investigational higher dose nusinersen regimen to advance the treatment of SMA. Biogen plans to submit for regulatory approval […]
Read More ›Congressional Action Recognizes Unmet Needs of SMA Community
This week, a key congressional committee recognized the unmet, everyday living needs of individuals with spinal muscular atrophy (SMA) by urging the National Institutes of […]
Read More ›Biogen Releases SMA Community Letter
On April 19, 2024, Biogen released an update to the SMA community sharing recent data presentations from Spring conferences and latest updates from their SPINRAZA® […]
Read More ›Our History
Make today a breakthrough. Cure SMA was founded in 1984 to support the SMA community and fund research to find a cure. In our 40-year […]
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