Author name: Sarah McCall

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.

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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

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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 »

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

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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 individuals who use wheelchairs, including passengers with spinal muscular atrophy (SMA). Cure SMA attended the February 29th White House announcement and plans to submit public

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

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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 $100,000 Grant to Elana Molotsky, PhD, at the Johns Hopkins University School of Medicine Read More »

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

Leave a Comment / Front Page News, Our Impact, Research /

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 Melissa Bowerman, PhD, at Keele University Read More »

New Report Highlights Caregiving Challenges of Individuals with Spinal Muscular Atrophy

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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 state and federal actions to help address their caregiver needs. Stuck Inside: A National Report on Caregiving for Individuals with SMA features numerous first-person experiences and quotes highlighting the essential

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

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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 helping individuals and families throughout the island. In late January, the Puerto Rico community came together to celebrate their new chapter through Summit of Strength and Walk-n-Roll events, with over

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