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
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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 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
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
Rare Disease Month 2025: Driving Progress Through Research Read More »
Cure SMA warmly invites you to join us at one of our 2025 Walk-n-Roll events—where community meets impact! This year, for the first time, you can register and start fundraising for both spring and fall events right now. Walk-n-Roll isn’t just a fundraiser; it’s a celebration of progress, hope, and togetherness. It’s a chance to
All 2025 Walk-n-Roll Events Are Open Read More »
In the RESILIENT SMA study, Biohaven announced today that taldefgrobep alpha showed clinically meaningful improvements in motor function at all time points on the Motor Function Measurement-32 scale (MFM-32), but the treatment arm did not statistically separate on the primary outcome at Week 48 compared to the placebo+standard of care (SOC) group. Biohaven plans to
Table of Contents What is SMA How Common is SMA Types of SMA Causes of SMA Carrier Testing Symptoms of SMA Diagnosis SMA Newborn Screening Treatment for SMA Outlook / Prognosis Living with SMA Latest Updates on SMA SMA Research SMA Resources and Programs Clinical Trials and Studies Common SMA Questions Next Steps for SMA
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