Search Results: Evrysdi
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 […]
Read More ›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 […]
Read More ›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, […]
Read More ›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 […]
Read More ›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 […]
Read More ›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 […]
Read More ›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 […]
Read More ›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 […]
Read More ›Cure SMA Releases Updated Drug Pipeline
We’ve recently released an update to the SMA drug pipeline. This latest version includes: 20 active programs, including three approved therapies. 10 pharmaceutical partners. 12 […]
Read More ›Scholar Rock Shares SMA Community Letter and Announces Design of Phase 3 SAPPHIRE Clinical Trial
Dear SMA Community, We are excited to announce the design of SAPPHIRE, a Phase 3 clinical trial to further evaluate the safety and efficacy of […]
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