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 […]
Cure SMA Celebrates Dr. Mary Schroth as she Retires as Chief Medical Officer Read More »
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
New Advocacy Resource: Explore the Cure SMA State and Local Toolkit! Read More »
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
Understanding Gene Therapy: A Cure SMA Video Read More »
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,
Double Your Power, Double Our Progress: Help Cure SMA Unlock $150,000 for SMA Research! Read More »
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
Last Chance to Participate in the Annual Cure SMA Community Update Survey Read More »
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
Novartis Gene Therapies Releases Spring SMA Community Update Letter Read More »
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
Check Out Cure SMA’s 2024 Annual State of SMA Report Read More »
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
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). This helps the FDA make patient-centered decisions about new SMA drugs. This is a critical time for patient input on SMA treatment, as a variety of new “add-on” therapies and
“Evrysdi has robust potential to modify the SMA disease trajectory, and has already been used to treat thousands of patients to date. This approval marks another significant step forward,” said Levi Garraway, M.D., Ph.D., Genentech’s Chief Medical Officer and Head of Global Product Development. “The Evrysdi tablet combines established efficacy with convenience, providing an
