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. […]
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
Apitegromab Linked to Improved Outcomes for Those with Spinal Muscular Atrophy Read More »
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.
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
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
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 people. Yet, the SMA community is part of a greater community, the rare disease community, that comprises of 300 million people worldwide. Together, we create
Celebrate Rare Disease Day with Cure SMA Read More »
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
Cure SMA Applauds DOT Proposal to Make Air Travel Safer for Wheelchair Users Read More »
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.
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 »
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
New Report Highlights Caregiving Challenges of Individuals with Spinal Muscular Atrophy Read More »
