Search Results: Zolgensma
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. […]
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 […]
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 […]
Drug Discovery
Make today a breakthrough. Drug discovery takes what we’ve learned about the causes and biology of spinal muscular atrophy (SMA) and utilizes it for basic […]
Positive New Data for Genentech’s Evrysdi in Largest Trial Ever Undertaken in Patients With Previously-Treated Spinal Muscular Atrophy (SMA)
Genentech, announced earlier this month new two-year data from the JEWELFISH study evaluating Evrysdi® (risdiplam) in people with Type 1, 2 or 3 SMA aged 6 […]