Front Page News
Cure SMA Celebrates Dr. Mary Schroth as she Retires as Chief Medical Officer
We are honored to celebrate the incredible contributions of Dr. Mary Schroth, who is retiring after seven years as our Chief Medical Officer and after […]
Read More ›New Advocacy Resource: Explore the Cure SMA State and Local Toolkit!
Make an Impact in Your State and Community Cure SMA is proud to announce the launch of our brand-new State and Local Advocacy Toolkit, now […]
Read More ›Understanding Gene Therapy: A Cure SMA Video
A drug’s mechanism of action, or MOA, refers to how a drug or other substance produces an effect in the body. Understanding a drug’s mechanism […]
Read More ›Double Your Power, Double Our Progress: Help Cure SMA Unlock $150,000 for SMA Research!
During our Nunemaker Family Research Community Challenge, all donations will be matched up to $150,000! Cure SMA is proud to announce the launch of our […]
Read More ›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 […]
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