March 2024

Government Funding Bill Supports New SMA Research to Address Unmet Needs

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Cure SMA’s year-long advocacy campaign to educate Congress and the Administration about the unmet needs of individuals with spinal muscular atrophy (SMA) has concluded with a major legislative victory. This weekend, the President signed into law a government funding bill that included a provision in support of new SMA research at the National Institutes of […]

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Cure SMA Awards $114,000 Grant to Michael Tellier, PhD, at the University of Leicester in the United Kingdom

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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 then ranks the submitted proposals on both their scientific merit and relevance to Cure SMA’s research priorities. Funding is awarded to the highest ranked projects.

Cure SMA Awards $114,000 Grant to Michael Tellier, PhD, at the University of Leicester in the United Kingdom Read More »

Apitegromab Linked to Improved Outcomes for Those with Spinal Muscular Atrophy

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The study included 58 individuals with SMA types 2 and 3 who were treated with IV apitegromab for 52 weeks. At 36 months, treatment resulted in statistically significant improvements in motor function and mobility. Earlier this month, Scholar Rock shared that treatment with apitegromab was linked to improved muscle strength and range of motion, while

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Cure SMA Awards $150,000 Grant to Allison Ebert, PhD, at The Medical College of Wisconsin

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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 then ranks the submitted proposals on both their scientific merit and relevance to Cure SMA’s research priorities. Funding is awarded to the highest ranked projects.

Cure SMA Awards $150,000 Grant to Allison Ebert, PhD, at The Medical College of Wisconsin Read More »

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

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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 reported RESPOND efficacy results showing improved motor function in most participants treated with SPINRAZA after gene therapy Today Biogen, Inc. announced interim 6-month biomarker data

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

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

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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 children treated in previous clinical studies1-6 Nearly all patients treated maintained or improved motor milestones after 52 weeks, with most switching to the one-time gene

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

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