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
What is Spinal Muscular Atrophy Read More »
“There is an endearing tenderness in the love of a mother to a son that transcends all other affections of the heart,” said Washington Irvine. When watching River Rolle wriggle in his mother Dee’s lap while he sweetly places his hand on her chin and looks into her eyes, there is nothing more apparent than
Community Spotlight: Dee and River Rolle Read More »
In Sarah Manuel’s career as a school psychologist, she mentors elementary, middle and high school students as they navigate everyday challenges in life and education. Though it took time and self-acceptance, she is confident that living with SMA helps her relate to students on a deeper level. “Just because there are limits that a diagnosis
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This week, bills to address everyday challenges faced by individuals with spinal muscular atrophy (SMA) took important steps forward in the U.S. Congress. A key aviation bill that includes a section that will make air travel safer and more accessible for passengers with disabilities, including SMA, was approved by the U.S. House of Representatives by
Big Week in Washington for Cure SMA-Supported Legislation Read More »
Nieves and Valerie Gomez’s SMA story started in 2013, when their son Liam Taylor Gomez was born with SMA Type 1. Liam passed away at six months of age, and at that time Nieves made a commitment to dedicate the rest of his life to healthcare and helping others. Since then, he has worked in
Donor Spotlight: Nieves and Valerie Gomez Read More »
