May 2025

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 for a variety of reasons, including, but not limited to: Improved Understanding: knowing how a drug works can help individuals and families to feel more […]

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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, an incredible opportunity to double your impact in our efforts to support spinal muscular atrophy (SMA) research. Thanks to the generosity of the Nunemaker Family,

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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 SMA and their families bring a unique perspective that, collectively, helps us adapt to the changing landscape of SMA. Data from the survey also informs Cure SMA’s advocacy agenda and

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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 voices in their work. Read the full community update letter here. “As winter turns to spring, we want to recognize that we have had an

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