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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 of action (MOA) is crucial […]
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 Nunemaker Family Research Community Challenge, […]
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 SMA and their families bring […]
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 center patient, family, and community […]
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, showcasing data through 2024. The […]
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 that is being developed to […]
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