Search Results: Evrysdi
Research Study Opportunities
Make today a breakthrough. By participating in a research study, you will help the scientific and research communities accelerate therapy development for spinal muscular atrophy […]
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, […]
SMA Awareness Month
SMA Awareness Month 2025 Spinal muscular atrophy (SMA) is a progressive neurodegenerative disease that affects the motor nerve cells in the spinal cord and impacts […]
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 […]
Thank You for a Wonderful 2022 Annual SMA Conference!
Thank you to everyone who attended the 2022 Annual SMA Conference at the Disneyland Hotel in Anaheim, California! It was an impactful weekend of opportunities […]