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Last Chance to Participate in the Annual Cure SMA Community Update Survey
Since 2017, the Cure SMA Community Update Survey has collected data and information on the SMA community’s experiences and unmet needs. Every single individual with […]
Read More ›Novartis Gene Therapies Releases Spring SMA Community Update Letter
Novartis Gene Therapies recently released an update on their continued collaboration with the SMA community through events, advisory boards, clinical insights, and ongoing efforts to […]
Read More ›Check Out Cure SMA’s 2024 Annual State of SMA Report
Check Out Cure SMA’s 2024 Annual State of SMA Report Cure SMA is pleased to announce the launch of our next State of SMA report, […]
Read More ›FDA Grants Priority Review for Biologics License Application (BLA) for Apitegromab as a Treatment for Spinal Muscular Atrophy
Today, Scholar Rock announced that the U.S. Food and Drug Administration (FDA) has accepted its Biologics License Application (BLA) for apitegromab, an investigational muscle-targeted treatment […]
Read More ›SMA Community Risk Tolerance Update: Comparison of 2022 and 2017 SMA Risk/Benefit Survey Data
One of Cure SMA’s top priorities is to relay the SMA community’s treatment experiences and preferences to the United States Food and Drug Administration (FDA). […]
Read More ›FDA Approves Genentech’s Evrysdi Tablet as First and Only Tablet for Spinal Muscular Atrophy (SMA)
“Evrysdi has robust potential to modify the SMA disease trajectory, and has already been used to treat thousands of patients to date. This approval […]
Read More ›Cure SMA Launches Advocacy Campaign to Secure New Federal Research for SMA
To address the chronic health needs of individuals with spinal muscular atrophy (SMA), Cure SMA and the SMA community have launched a new advocacy campaign […]
Read More ›Rare Disease Month 2025: Driving Progress Through Research
February marks Rare Disease Month, a time to amplify the voices of the 300 million people worldwide living with a rare disease-including the individuals in […]
Read More ›Scholar Rock Submits Biologics License Application (BLA) to the U.S. FDA for Apitegromab as a Treatment for Patients with Spinal Muscular Atrophy (SMA)
“With the strength of our Phase 3 data as the foundation of our submission, we look forward to continuing to work closely with the […]
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 […]
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