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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 […]

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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 […]

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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 […]

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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 […]

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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 […]

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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 […]

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New Report Highlights Caregiving Challenges of Individuals with Spinal Muscular Atrophy

February 16, 2024
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Cure SMA released a national report on National Caregivers Day that chronicles the caregiving challenges experienced by individuals with spinal muscular atrophy (SMA) and recommends […]

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Cure SMA is Thrilled to Announce the Establishment of our New Chapter in Puerto Rico!

February 7, 2024
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Led by Keishla Rolon, Ramon Rivera Vega, Elizabeth Serrano, Natasha Santiago, and Brenda Luciano, Cure SMA’s Puerto Rico chapter has already been hard at work […]

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Spring 2024 Walk-n-Roll Registration is NOW OPEN!

January 18, 2024
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Cure SMA is excited to officially open registration for our Spring 2024 Walk-n-Roll events! By participating in our Walk-n-Roll, you can make a real difference in […]

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100% of States Now Screening Newborns for SMA

January 3, 2024
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You did it!   We’ve now reached our goal of 100 percent newborn screening of spinal muscular atrophy (SMA) in all 50 states with the […]

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