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New Biomarker Data Add Further Evidence Supporting the Potential Benefit of SPINRAZA® (nusinersen) in Infants and Toddlers with Unmet Clinical Needs after Gene Therapy

March 6, 2024
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  New data from the RESPOND study show that neurofilament levels, an indicator of neurodegeneration, were reduced in nearly all study participants treated with SPINRAZA Reductions in biomarker complement previously […]

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Novartis Presents New Data on Safety and Efficacy of Zolgensma, Including Maintained and Improved Motor Milestones in Older and Heavier Children with SMA

March 4, 2024
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  The SMART study supplements a growing body of evidence on the use of Zolgensma in a patient population older and heavier (1.5 – 9.1 years of age) than the […]

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Celebrate Rare Disease Day with Cure SMA

February 29, 2024
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Today is Rare Disease Day and the rarest day of all, as this year’s occurrence takes place on February 29! In the U.S., spinal muscular atrophy (SMA) affects nearly 9,000 […]

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Cure SMA Applauds DOT Proposal to Make Air Travel Safer for Wheelchair Users

February 29, 2024
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Today, on Rare Disease Day, U.S. Transportation Secretary Pete Buttigieg and White House officials announced actions the U.S. Department of Transportation (DOT) plans to take to improve air safety for […]

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Cure SMA Awards $100,000 Grant to Elana Molotsky, PhD, at the Johns Hopkins University School of Medicine

February 26, 2024
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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 […]

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Cure SMA Awards $150,000 Grant to Melissa Bowerman, PhD, at Keele University

February 21, 2024
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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 […]

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