Search Results: spinraza
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 […]
Read More ›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 […]
Read More ›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 2024 Coming Soon in August 2024 Spinal muscular atrophy (SMA) is a progressive neurodegenerative disease that affects the motor nerve cells […]
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 ›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 […]
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