Search Results: spinraza
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) […]
Cure SMA Awards $75,000 Grant to Lyndsay Murray, PhD, University of Edinburgh
Lyndsay Murray, PhD, was awarded $75,000 for her research project, “Evaluating the impact of sequential versus simultaneous administration of SMN-inducing compounds on motor unit recovery […]
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. […]
FAQs & Glossary
Make today a breakthrough. Learn more about spinal muscular atrophy (SMA) by reading our frequently asked questions and glossary of common terms. If you still […]
Cure SMA Awards $150,000 Grant to Alberto Kornblihtt, PhD, Universidad de Buenos Aires
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 […]
Newborn Screening for SMA
Make today a breakthrough. Evidence shows that early diagnosis through newborn screening and early intervention with available treatments lead to better outcomes. This is especially […]
Find a Treatment Center
Make today a breakthrough. Our online location finder is designed to help individuals with spinal muscular atrophy (SMA) locate treatment and medical care in your […]
Spinal Muscular Atrophy Treatment
Make today a breakthrough. There are several (SMA) spinal muscular atrophy treatments approved. Each individual or family must make treatment decisions based on your needs, […]
Approaches to Drug Development
Make today a breakthrough. Due to a mutation in the survival motor neuron gene 1 (SMN1), individuals with spinal muscular atrophy (SMA) don’t produce survival […]
SMA Drug Pipeline
Make today a breakthrough. The SMA Drug Pipeline is how Cure SMA evaluates the success of our spinal muscular atrophy (SMA) research program. It identifies […]