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Understanding Gene Therapy: A Cure SMA Video

May 19, 2025
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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 […]

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Double Your Power, Double Our Progress: Help Cure SMA Unlock $150,000 for SMA Research!

May 19, 2025
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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, […]

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Last Chance to Participate in the Annual Cure SMA Community Update Survey

May 16, 2025
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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 […]

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Novartis Gene Therapies Releases Spring SMA Community Update Letter

May 5, 2025
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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 […]

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Check Out Cure SMA’s 2024 Annual State of SMA Report

March 31, 2025
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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 […]

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FDA Grants Priority Review for Biologics License Application (BLA) for Apitegromab as a Treatment for Spinal Muscular Atrophy

March 25, 2025
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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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