This week, Cure SMA urged the U.S. Department of Transportation (DOT) to strengthen and quickly implement its proposed rule on ensuring safe accommodations for air travelers with spinal muscular atrophy (SMA) and other disabilities who use wheelchairs. In the 9-page letter addressed to Secretary Pete Buttigieg, Cure SMA highlighted critical issues faced by individuals with […]
Accessible Air Travel Update – Next Steps Read More »
Cure SMA is pleased to announce the release of new resources to support our community’s ability to navigate the evolving landscape of SMA clinical trials. These resources are intended to build knowledge in clinical trial procedures and preparation, to enhance awareness regarding actively recruiting clinical trials and sites, and to empower our community in their
Cure SMA Launches New Resources to Enhance Clinical Trial Access Read More »
Cure SMA is pleased to announce the launch of an expanded Phase 9 of our SMA Industry Collaboration. The SMA Industry Collaboration is a multi-faceted partnership that brings together pharmaceutical companies, SMA Europe, Cure SMA, and other nonprofit organizations to share information, ideas, and data to benefit the broader SMA community. Through the Industry Collaboration,
Cure SMA Announces Expanded Phase 9 of SMA Industry Collaboration Read More »
For the past seven years, the Cure SMA Community Update Survey has collected data and information on the SMA community’s experiences and daily challenges. Every single individual with SMA and their families bring a unique perspective that, collectively, help us adapt to the changing landscape of SMA. Data from the survey also informs Cure SMA’s
Participate in the Annual Cure SMA Community Update Survey – Open Now! 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.
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
Government Funding Bill Supports New SMA Research to Address Unmet Needs 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.
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
