Author name: Zach Galati

Cure SMA Awards $150,000 Grant to Charlotte Sumner, MD, at Johns Hopkins University School of Medicine

Each year, Cure SMA invites scientists from around the world to submit funding proposals for basic research projects that address specific unanswered questions in SMA biology. Our Scientific Advisory Board then ranks the submitted proposals on both their scientific merit and relevance to Cure SMA’s research priorities. Funding is awarded to the highest ranked projects. […]

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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 many decades of service and care for our community.   Honoring Dr. Mary Schroth’s Legacy   For several decades, Dr. Mary Schroth has been a driving force in advancing clinical

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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 available on the Advocacy webpage! This interactive resource empowers individuals and families in the SMA community to take action and drive change at the local and state level. Whether you’re

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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 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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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 annual data report highlights data from Cure SMA’s three databases: a patient-reported database with data from over 11,000 impacted individuals worldwide that also incorporates longitudinal

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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 provide clinically meaningful improvement in motor function for people living with spinal muscular atrophy (SMA) who are receiving an SMN-targeted treatment. The FDA will review

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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 the U.S. impacted by spinal muscular atrophy (SMA). Throughout this month, and especially during Rare Disease Week (starting February 23rd), we’re committed to advancing research, raising awareness, and advocating for

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