Search Results: Zolgensma
Cure SMA Awards $150,000 Grant to Anton Blatnik, PhD, at Case Western Reserve University
Each year, Cure SMA invites scientists from around the world to submit funding proposals for basic research projects that address specific unanswered questions in spinal […]
Itvisma®
Itvisma® Itvisma® is an FDA approved treatment for spinal muscular atrophy (SMA). It is a type of treatment referred to as gene therapy or gene […]
Novartis Gene Therapies Releases Spring SMA Community Letter
Novartis Gene Therapies recently released an update to the SMA community related to clinical trials and long term follow up studies of Zolgensma, in addition […]
New Biomarker Data Add Further Evidence Supporting the Potential Benefit of SPINRAZA® (nusinersen) in Infants and Toddlers with Unmet Clinical Needs after Gene Therapy
New data from the RESPOND study show that neurofilament levels, an indicator of neurodegeneration, were reduced in nearly all study participants treated with SPINRAZA […]
Our History
Make today a breakthrough. Cure SMA was founded in 1984 to support the SMA community and fund research to find a cure. In our 40-year […]
SMA Scientists and Clinicians from Around the World Gather at the 27th Annual SMA Research & Clinical Care Meeting
At the end of June, SMA researchers and clinicians from around the world met in Orlando, Florida, for the 2023 Annual SMA Research & Clinical […]
New Data at Cure SMA Highlight Potential Benefit of SPINRAZA® (nusinersen) in Infants and Toddlers with Unmet Clinical Needs After Gene Therapy
Biogen Inc. announced new SPINRAZA® (nusinersen) data aimed at answering critical questions for the spinal muscular atrophy (SMA) community. The data were presented at the SMA […]
Cure SMA International Resources
Welcome and thank you for reaching out to Cure SMA! The diagnosis of spinal muscular atrophy (SMA) can be confusing, scary, frustrating, pretty much the […]
Biogen Announces New Updates Across its SMA Research Program at 2023 MDA Conference
First patient treated in the ASCEND study evaluating the potential benefit of investigational higher dose nusinersen in children, teens and adults previously treated with Evrysdi® (risdiplam) […]
Therapeutic Approaches
Make today a breakthrough. One of the strengths of Cure SMA’s drug discovery program is how we address spinal muscular atrophy (SMA) from multiple angles. […]